Volume 18

Molecular Therapy

Volume 18

1

A Comparison of the Growth Responses Following Intramuscular GHRH Plasmid Administration Versus Daily Growth Hormone Injections in Young Pigs

Khan, Amir S, Draghia-Akli, Ruxandra, Shypailo, Roman J, Ellis, Kenneth I, Mersmann, Harry, Fiorotto, Marta L

Journal:

Molecular Therapy

Year:

2009

Language:

english

File:

PDF, 178 KB

english, 2009

2

Adipose-derived Stem Cells as Therapeutic Delivery Vehicles of an Oncolytic Virus for Glioblastoma

Josiah, Darnell T, Zhu, Dongqin, Dreher, Fernanda, Olson, John, McFadden, Grant, Caldas, Hannah

Journal:

Molecular Therapy

Year:

2009

Language:

english

File:

PDF, 596 KB

english, 2009

3

Complete Genetic Correction of iPS Cells From Duchenne Muscular Dystrophy

Kazuki, Yasuhiro, Hiratsuka, Masaharu, Takiguchi, Masato, Osaki, Mitsuhiko, Kajitani, Naoyo, Hoshiya, Hidetoshi, Hiramatsu, Kei, Yoshino, Toko, Kazuki, Kanako, Ishihara, Chie

Journal:

Molecular Therapy

Year:

2009

Language:

english

File:

PDF, 892 KB

english, 2009

4

Intracellular Trafficking and Decondensation Kinetics of Chitosan–pDNA Polyplexes

Thibault, Marc, Nimesh, Surendra, Lavertu, Marc, Buschmann, Michael D

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 860 KB

english, 2010

5

RNAi-mediated CCR5 Silencing by LFA-1-targeted Nanoparticles Prevents HIV Infection in BLT Mice

Kim, Sang-Soo, Peer, Dan, Kumar, Priti, Subramanya, Sandesh, Wu, Huaquan, Asthana, Deshratan, Habiro, Katsuyoshi, Yang, Yong-Guang, Manjunath, N, Shimaoka, Motomu, Shankar, Premlata

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 283 KB

english, 2010

6

The Chase for the RIG-I Ligand—Recent Advances

Schlee, Martin, Hartmann, Gunther

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 194 KB

english, 2010

7

Inhibition of Multiple Protective Signaling Pathways and Ad.5/3 Delivery Enhances mda-7/IL-24 Therapy of Malignant Glioma

Hamed, Hossein A, Yacoub, Adly, Park, Margaret A, Eulitt, Patrick J, Dash, Rupesh, Sarkar, Devanand, Dmitriev, Igor P, Lesniak, Maciej S, Shah, Khalid, Grant, Steven, Curiel, David T, Fisher, Paul B,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 549 KB

english, 2010

8

miR-122 Continues to Blaze the Trail for MicroRNA Therapeutics

Haussecker, Dirk, Kay, Mark A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 213 KB

english, 2010

9

Reactive Bone Marrow Stromal Cells Attenuate Systemic Inflammation via sTNFR1

Yagi, Hiroshi, Soto-Gutierrez, Alejandro, Navarro-Alvarez, Nalu, Nahmias, Yaakov, Goldwasser, Yoni, Kitagawa, Yuko, Tilles, Arno W, Tompkins, Ronald G, Parekkadan, Biju, Yarmush, Martin L

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 938 KB

english, 2010

10

Targeting Improves MSC Treatment of Inflammatory Bowel Disease

Ko, In Kap, Kim, Byung-Gyu, Awadallah, Amad, Mikulan, Jenifer, Lin, Paul, Letterio, John J, Dennis, James E

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.69 MB

english, 2010

11

Dystrophin Delivery to Muscles of mdx Mice Using Lentiviral Vectors Leads to Myogenic Progenitor Targeting and Stable Gene Expression

Kimura, En, Li, Sheng, Gregorevic, Paul, Fall, Brent M, Chamberlain, Jeffrey S

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1013 KB

english, 2010

12

Orally Administered Bifidobacteria as Vehicles for Delivery of Agents to Systemic Tumors

Cronin, Michelle, Morrissey, David, Rajendran, Simon, El Mashad, Shereen M, van Sinderen, Douwe, O'Sullivan, Gerald C, Tangney, Mark

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 276 KB

english, 2010

13

Engineered Newcastle Disease Virus as an Improved Oncolytic Agent Against Hepatocellular Carcinoma

Altomonte, Jennifer, Marozin, Sabrina, Schmid, Roland M, Ebert, Oliver

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 772 KB

english, 2010

14

Systemic Delivery of Synthetic MicroRNA-16 Inhibits the Growth of Metastatic Prostate Tumors via Downregulation of Multiple Cell-cycle Genes

Takeshita, Fumitaka, Patrawala, Lubna, Osaki, Mitsuhiko, Takahashi, Ryou-u, Yamamoto, Yusuke, Kosaka, Nobuyoshi, Kawamata, Masaki, Kelnar, Kevin, Bader, Andreas G., Brown, David, Ochiya, Takahiro

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 373 KB

english, 2010

15

Zinc-finger Nucleases as a Novel Therapeutic Strategy for Targeting Hepatitis B Virus DNAs

Cradick, Thomas J, Keck, Kathy, Bradshaw, Shannon, Jamieson, Andrew C, McCaffrey, Anton P

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 484 KB

english, 2010

16

Lentivirus-mediated Reprogramming of Somatic Cells in the Absence of Transgenic Transcription Factors

Kane, Nicole M, Nowrouzi, Ali, Mukherjee, Sayandip, Blundell, Michael P, Greig, Jenny A, Lee, Wai Kwong, Houslay, Miles D, Milligan, Graeme, Mountford, Joanne C, von Kalle, Christof, Schmidt, Manfred,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 593 KB

english, 2010

17

First-in-human Mutation-targeted siRNA Phase Ib Trial of an Inherited Skin Disorder

Leachman, Sancy A, Hickerson, Robyn P, Schwartz, Mary E, Bullough, Emily E, Hutcherson, Stephen L, Boucher, Kenneth M, Hansen, C David, Eliason, Mark J, Srivatsa, G Susan, Kornbrust, Douglas J, Smith,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 264 KB

english, 2010

18

Recent Advances in Lentiviral Vector Development and Applications

Mátrai, Janka, Chuah, Marinee KL, VandenDriessche, Thierry

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 659 KB

english, 2010

19

A Novel Nanoparticle Drug Delivery System: The Anti-inflammatory Activity of Curcumin Is Enhanced When Encapsulated in Exosomes

Sun, Dongmei, Zhuang, Xiaoying, Xiang, Xiaoyu, Liu, Yuelong, Zhang, Shuangyin, Liu, Cunren, Barnes, Stephen, Grizzle, William, Miller, Donald, Zhang, Huang-Ge

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 474 KB

english, 2010

20

A Transposon and Transposase System for Human Application

Hackett, Perry B, Largaespada, David A, Cooper, Laurence JN

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 833 KB

english, 2010

21

Targeted Delivery of siRNA to Macrophages for Anti-inflammatory Treatment

Kim, Sang-Soo, Ye, Chunting, Kumar, Priti, Chiu, Isaac, Subramanya, Sandesh, Wu, Haoquan, Shankar, Premlata, Manjunath, N

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 469 KB

english, 2010

22

A Short Pulse of IL-4 Delivered by DCs Electroporated With Modified mRNA Can Both Prevent and Treat Autoimmune Diabetes in NOD Mice

Creusot, Rémi J, Chang, Pearl, Healey, Don G, Tcherepanova, Irina Y, Nicolette, Charles A, Fathman, C Garrison

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 442 KB

english, 2010

23

Advanced Phase I/II Studies of Targeted Gene Delivery In Vivo: Intravenous Rexin-G for Gemcitabine-resistant Metastatic Pancreatic Cancer

Chawla, Sant P, Chua, Victoria S, Fernandez, Lita, Quon, Dorris, Blackwelder, William C, Gordon, Erlinda M, Hall, Frederick L

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 466 KB

english, 2010

24

The Use of Inhibitors to Study Endocytic Pathways of Gene Carriers: Optimization and Pitfalls

Vercauteren, Dries, Vandenbroucke, Roosmarijn E, Jones, Arwyn T, Rejman, Joanna, Demeester, Joseph, De Smedt, Stefaan C, Sanders, Niek N, Braeckmans, Kevin

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 596 KB

english, 2010

25

Adipose Tissue–derived Mesenchymal Stem Cells Expressing Prodrug-converting Enzyme Inhibit Human Prostate Tumor Growth

Cavarretta, Ilaria T, Altanerova, Veronika, Matuskova, Miroslava, Kucerova, Lucia, Culig, Zoran, Altaner, Cestmir

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 398 KB

english, 2010

26

Toward Gene Therapy for Cystic Fibrosis Using a Lentivirus Pseudotyped With Sendai Virus Envelopes

Mitomo, Katsuyuki, Griesenbach, Uta, Inoue, Makoto, Somerton, Lucinda, Meng, Cuixiang, Akiba, Eiji, Tabata, Toshiaki, Ueda, Yasuji, Frankel, Gad M, Farley, Raymond, Singh, Charanjit, Chan, Mario, Munk

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 867 KB

english, 2010

27

Lentivirus Immobilization to Nanoparticles for Enhanced and Localized Delivery From Hydrogels

Shin, Seungjin, Shea, Lonnie D

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 380 KB

english, 2010

28

Therapeutic and Tumor-specific Immunity Induced by Combination of Dendritic Cells and Oncolytic Adenovirus Expressing IL-12 and 4-1BBL

Huang, Jing-Hua, Zhang, Song-Nan, Choi, Kyung-Ju, Choi, Il-Kyu, Kim, Joo-Hang, Lee, Mingul, Kim, Hoguen, Yun, Chae-Ok

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 638 KB

english, 2010

29

Intelligent Design: Combination Therapy With Oncolytic Viruses

Ottolino-Perry, Kathryn, Diallo, Jean-Simon, Lichty, Brian D, Bell, John C, Andrea McCart, J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 264 KB

english, 2010

30

Verapamil Enhances the Antitumoral Efficacy of Oncolytic Adenoviruses

Gros, Alena, Puig, Cristina, Guedan, Sonia, Rojas, Juan José, Alemany, Ramon, Cascallo, Manel

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 523 KB

english, 2010

31

Combined Paracrine and Endocrine AAV9 mediated Expression of Hepatocyte Growth Factor for the Treatment of Renal Fibrosis

Schievenbusch, Stephanie, Strack, Ingo, Scheffler, Melanie, Nischt, Roswitha, Coutelle, Oliver, Hösel, Marianna, Hallek, Michael, Fries, Jochen WU, Dienes, Hans-Peter, Odenthal, Margarete, Büning, Hil

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 499 KB

english, 2010

32

Gene Transfer Efficiency and Genome-Wide Integration Profiling of Sleeping Beauty, Tol2, and PiggyBac Transposons in Human Primary T Cells

Huang, Xin, Guo, Hongfeng, Tammana, Syam, Jung, Yong-Chul, Mellgren, Emil, Bassi, Preetinder, Cao, Qing, Tu, Zheng Jin, Kim, Yeong C, Ekker, Stephen C, Wu, Xiaolin, Wang, San Ming, Zhou, Xianzheng

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 711 KB

english, 2010

33

Case Report of a Serious Adverse Event Following the Administration of T Cells Transduced With a Chimeric Antigen Receptor Recognizing ERBB2

Morgan, Richard A, Yang, James C, Kitano, Mio, Dudley, Mark E, Laurencot, Carolyn M, Rosenberg, Steven A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 264 KB

english, 2010

34

A Phase I Study of Telomerase-specific Replication Competent Oncolytic Adenovirus (Telomelysin) for Various Solid Tumors

Nemunaitis, John, Tong, Alex W, Nemunaitis, Michael, Senzer, Neil, Phadke, Anagha P, Bedell, Cynthia, Adams, Ned, Zhang, Yu-An, Maples, Phillip B, Chen, Salina, Pappen, Beena, Burke, James, Ichimaru,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 231 KB

english, 2010

35

siRNA Nanoparticle Functionalization of Nanostructured Scaffolds Enables Controlled Multilineage Differentiation of Stem Cells

Andersen, Morten Ø, Nygaard, Jens V, Burns, Jorge S, Raarup, Merete K, Nyengaard, Jens R, Bünger, Cody, Besenbacher, Flemming, Howard, Kenneth A, Kassem, Moustapha, Kjems, Jørgen

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 500 KB

english, 2010

36

Nanoparticles Modified With Tumor-targeting scFv Deliver siRNA and miRNA for Cancer Therapy

Chen, Yunching, Zhu, Xiaodong, Zhang, Xiaoju, Liu, Bin, Huang, Leaf

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 609 KB

english, 2010

37

Intramuscular Transplantation of Human Postnatal Myoblasts Generates Functional Donor-Derived Satellite Cells

Skuk, Daniel, Paradis, Martin, Goulet, Marlyne, Chapdelaine, Pierre, Rothstein, David M, Tremblay, Jacques P

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.08 MB

english, 2010

38

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

Heemskerk, Hans, de Winter, Christa, van Kuik, Petra, Heuvelmans, Niki, Sabatelli, Patrizia, Rimessi, Paola, Braghetta, Paola, van Ommen, Gert-Jan B, de Kimpe, Sjef, Ferlini, Alessandra, Aartsma-Rus,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 481 KB

english, 2010

39

Macrophage-mediated GDNF Delivery Protects Against Dopaminergic Neurodegeneration: A Therapeutic Strategy for Parkinson's Disease

Biju, KC, Zhou, Qing, Li, Guiming, Imam, Syed Z, Roberts, James L, Morgan, William W, Clark, Robert A, Li, Senlin

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 607 KB

english, 2010

40

Inhibition of Tumor Growth and Metastasis by a Combination of Escherichia coli–mediated Cytolytic Therapy and Radiotherapy

Jiang, Sheng-Nan, Phan, Thuy X, Nam, Taek-Keun, Nguyen, Vu H, Kim, Hyung-Seok, Bom, Hee-Seung, Choy, Hyon E, Hong, Yeongjin, Min, Jung-Joon

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 702 KB

english, 2010

41

piggyBac Transposon-mediated Long-term Gene Expression in Mice

Nakanishi, Hideyuki, Higuchi, Yuriko, Kawakami, Shigeru, Yamashita, Fumiyoshi, Hashida, Mitsuru

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 287 KB

english, 2010

42

A Vaccine Targeting Telomerase Enhances Survival of Dogs Affected by B-cell Lymphoma

Peruzzi, Daniela, Gavazza, Alessandra, Mesiti, Giuseppe, Lubas, George, Scarselli, Elisa, Conforti, Antonella, Bendtsen, Claus, Ciliberto, Gennaro, La Monica, Nicola, Aurisicchio, Luigi

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 316 KB

english, 2010

43

Comparative Analysis of Transposable Element Vector Systems in Human Cells

Grabundzija, Ivana, Irgang, Markus, Mátés, Lajos, Belay, Eyayu, Matrai, Janka, Gogol-Döring, Andreas, Kawakami, Koichi, Chen, Wei, Ruiz, Patricia, Chuah, Marinee K. L., VandenDriessche, Thierry, Izsvá

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 530 KB

english, 2010

44

Synergistic Interaction Between Oncolytic Viruses Augments Tumor Killing

Le Boeuf, Fabrice, Diallo, Jean-Simon, McCart, J Andrea, Thorne, Steve, Falls, Theresa, Stanford, Marianne, Kanji, Femina, Auer, Rebecca, Brown, Christopher W, Lichty, Brian D, Parato, Kelley, Atkins,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 563 KB

english, 2010

45

Potentiating Cancer Immunotherapy Using an Oncolytic Virus

Bridle, Byram W, Stephenson, Kyle B, Boudreau, Jeanette E, Koshy, Sandeep, Kazdhan, Natasha, Pullenayegum, Eleanor, Brunellière, Jérôme, Bramson, Jonathan L, Lichty, Brian D, Wan, Yonghong

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 399 KB

english, 2010

46

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

Simonelli, Francesca, Maguire, Albert M, Testa, Francesco, Pierce, Eric A, Mingozzi, Federico, Bennicelli, Jeannette L, Rossi, Settimio, Marshall, Kathleen, Banfi, Sandro, Surace, Enrico M, Sun, Junwe

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 415 KB

english, 2010

47

Particle Tracking of Intracellular Trafficking of Octaarginine-modified Liposomes: A Comparative Study With Adenovirus

Akita, Hidetaka, Enoto, Kaoru, Masuda, Tomoya, Mizuguchi, Hiroyuki, Tani, Tomomi, Harashima, Hideyoshi

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 631 KB

english, 2010

48

CNS-targeted LIF Expression Improves Therapeutic Efficacy and Limits Autoimmune-mediated Demyelination in a Model of Multiple Sclerosis

Slaets, Helena, Hendriks, Jerome JA, Van den Haute, Chris, Coun, Frea, Baekelandt, Veerle, Stinissen, Piet, Hellings, Niels

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 490 KB

english, 2010

49

Mesenchymal Stem Cells Expressing Osteogenic and Angiogenic Factors Synergistically Enhance Bone Formation in a Mouse Model of Segmental Bone Defect

Kumar, Sanjay, Wan, Chao, Ramaswamy, Girish, Clemens, Thomas L, Ponnazhagan, Selvarangan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 669 KB

english, 2010

50

Toward Brain Tumor Gene Therapy Using Multipotent Mesenchymal Stromal Cell Vectors

Bexell, Daniel, Scheding, Stefan, Bengzon, Johan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 319 KB

english, 2010

51

Chimeric Antigen Receptors Combining 4-1BB and CD28 Signaling Domains Augment PI3kinase/AKT/Bcl-XL Activation and CD8+ T Cell–mediated Tumor Eradication

Zhong, Xiao-Song, Matsushita, Maiko, Plotkin, Jason, Riviere, Isabelle, Sadelain, Michel

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 347 KB

english, 2010

52

Effects of Localized Neurotrophin Gene Expression on Spiral Ganglion Neuron Resprouting in the Deafened Cochlea

Wise, Andrew K, Hume, Clifford R, Flynn, Brianna O, Jeelall, Yogesh S, Suhr, Courtney L, Sgro, Beatrice E, O'Leary, Stephen J, Shepherd, Robert K, Richardson, Rachael T

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 987 KB

english, 2010

53

Persistent Expression of FLAG-tagged Micro dystrophin in Nonhuman Primates Following Intramuscular and Vascular Delivery

Rodino-Klapac, Louise R, Montgomery, Chrystal L, Bremer, William G, Shontz, Kimberly M, Malik, Vinod, Davis, Nancy, Sprinkle, Spencer, Campbell, Katherine J, Sahenk, Zarife, Clark, K Reed, Walker, Chr

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.42 MB

english, 2010

54

Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs

Haurigot, Virginia, Mingozzi, Federico, Buchlis, George, Hui, Daniel J, Chen, Yifeng, Basner-Tschakarjan, Etiena, Arruda, Valder R, Radu, Antoneta, Franck, Helen G, Wright, J Fraser, Zhou, Shangzhen,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 667 KB

english, 2010

55

Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy

Chandler, Randy J, Venditti, Charles P

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

56

Design and Evaluation of Doxorubicin-containing Microbubbles for Ultrasound-triggered Doxorubicin Delivery: Cytotoxicity and Mechanisms Involved

Lentacker, Ine, Geers, Bart, Demeester, Joseph, De Smedt, Stefaan C, Sanders, Niek N

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 692 KB

english, 2010

57

Neonatal Gene Therapy of Glycogen Storage Disease Type Ia Using a Feline Immunodeficiency Virus–based Vector

Grinshpun, Albert, Condiotti, Reba, N Waddington, Simon, Peer, Michael, Zeig, Eli, Peretz, Sima, Simerzin, Alina, Chou, Janice, Pann, Chi-Jiunn, Giladi, Hilla, Galun, Eithan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 418 KB

english, 2010

58

AAV8-mediated Gene Therapy Prevents Induced Biochemical Attacks of Acute Intermittent Porphyria and Improves Neuromotor Function

Yasuda, Makiko, Bishop, David F, Fowkes, Mary, Cheng, Seng H, Gan, Lin, Desnick, Robert J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 167 KB

english, 2010

59

Bone Marrow Mesenchymal Stem Cells Loaded With an Oncolytic Adenovirus Suppress the Anti-adenoviral Immune Response in the Cotton Rat Model

Ahmed, Atique U, Rolle, Cleo E, Tyler, Matthew A, Han, Yu, Sengupta, Sadhak, Wainwright, Derek A, Balyasnikova, Irina V, Ulasov, Ilya V, Lesniak, Maciej S

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 592 KB

english, 2010

60

VSV Oncolysis in Combination With the BCL-2 Inhibitor Obatoclax Overcomes Apoptosis Resistance in Chronic Lymphocytic Leukemia

Samuel, Sara, Tumilasci, Vanessa F, Oliere, Stephanie, Liên-Anh Nguyên, Thi, Shamy, April, Bell, John, Hiscott, John

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 472 KB

english, 2010

61

Targeting Localized Immune Suppression Within the Tumor Through Repeat Cycles of Immune Cell-oncolytic Virus Combination Therapy

Thorne, Stephen H, Liang, Wenchun, Sampath, Padma, Schmidt, Tobi, Sikorski, Rachel, Beilhack, Andreas, Contag, Christopher H

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 274 KB

english, 2010

62

Engineering of Human Pluripotent Stem Cells by AAV-mediated Gene Targeting

Khan, Iram F, Hirata, Roli K, Wang, Pei-Rong, Li, Yi, Kho, Jordan, Nelson, Angel, Huo, Yunwen, Zavaljevski, Maja, Ware, Carol, Russell, David W

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 427 KB

english, 2010

63

Intranodal Immunization With a Vaccinia Virus Encoding Multiple Antigenic Epitopes and Costimulatory Molecules in Metastatic Melanoma

Adamina, Michel, Rosenthal, Rachel, Weber, Walter P, Frey, Daniel M, Viehl, Carsten T, Bolli, Martin, Huegli, Rolf W, Jacob, Augustinus L, Heberer, Michael, Oertli, Daniel, Marti, Walter, Spagnoli, Gi

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 286 KB

english, 2010

64

IL-28B/IFN-λ3 Drives Granzyme B Loading and Significantly Increases CTL Killing Activity in Macaques

Morrow, Matthew P, Yan, Jian, Pankhong, Panyupa, Shedlock, Devon J, Lewis, Mark G, Talbott, Kendra, Toporovski, Roberta, Khan, Amir S, Sardesai, Niranjan Y, Weiner, David B

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 390 KB

english, 2010

65

The Pharmacological Chaperone 1-Deoxygalactonojirimycin Reduces Tissue Globotriaosylceramide Levels in a Mouse Model of Fabry Disease

Khanna, Richie, Soska, Rebecca, Lun, Yi, Feng, Jessie, Frascella, Michelle, Young, Brandy, Brignol, Nastry, Pellegrino, Lee, Sitaraman, Sheela A, Desnick, Robert J, Benjamin, Elfrida R, Lockhart, Davi

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 641 KB

english, 2010

66

Duration of Expression and Activity of Sleeping Beauty Transposase in mouse liver following hydrodynamic DNA delivery

Bell, Jason B, Aronovich, Elena L, Schreifels, Jeffrey M, Beadnell, Thomas C, Hackett, Perry B

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 346 KB

english, 2010

67

Polyelectrolyte Capsules-containing HIV-1 p24 and Poly I:C Modulate Dendritic Cells to Stimulate HIV-1-specific Immune Responses

De Haes, Winni, De Koker, Stefaan, Pollard, Charlotte, Atkinson, Derek, Vlieghe, Erika, Hoste, Jessy, Rejman, Joanna, De Smedt, Stefaan, Grooten, Johan, Vanham, Guido, Van Gulck, Ellen

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 311 KB

english, 2010

68

Treatment of Chronic Lymphocytic Leukemia With Genetically Targeted Autologous T Cells: Case Report of an Unforeseen Adverse Event in a Phase I Clinical Trial

Brentjens, Renier, Yeh, Raymond, Bernal, Yvette, Riviere, Isabelle, Sadelain, Michel

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 199 KB

english, 2010

69

MAPK Signal-integrating Kinase Controls Cap-independent Translation and Cell Type-specific Cytotoxicity of an Oncolytic Poliovirus

Goetz, Christian, Everson, Richard G, Zhang, Linda C, Gromeier, Matthias

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 786 KB

english, 2010

70

Novel RNA-based Strategies for Therapeutic Gene Silencing

Sibley, Christopher R, Seow, Yiqi, Wood, Matthew JA

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 394 KB

english, 2010

71

Rational Design of Immunostimulatory siRNAs

Gantier, Michael P, Tong, Stephen, Behlke, Mark A, Irving, Aaron T, Lappas, Martha, Nilsson, Ulrika W, Latz, Eicke, McMillan, Nigel AJ, Williams, Bryan RG

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 936 KB

english, 2010

72

Characterization of Genome Integrity for Oversized Recombinant AAV Vector

Dong, Biao, Nakai, Hiroyuki, Xiao, Weidong

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

73

Site-specific Gene Insertion Mediated by a Cre-loxP-carrying Lentiviral Vector

Michel, Gilles, Yu, Yin, Chang, Tammy, Yee, Jiing-Kuan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 378 KB

english, 2010

74

Expression of Dog Microdystrophin in Mouse and Dog Muscles by Gene Therapy

Pichavant, Christophe, Chapdelaine, Pierre, Cerri, Daniel G, Dominique, Jean-Christophe, Quenneville, Simon P, Skuk, Daniel, Kornegay, Joe N, Bizario, João CS, Xiao, Xiao, Tremblay, Jacques P

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.23 MB

english, 2010

75

Noninvasive Imaging of Lipid Nanoparticle–Mediated Systemic Delivery of Small-Interfering RNA to the Liver

Tao, Weikang, Davide, Joseph P, Cai, Mingmei, Zhang, Guo-Jun, South, Victoria J, Matter, Andrea, Ng, Bruce, Zhang, Ye, Sepp-Lorenzino, Laura

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 790 KB

english, 2010

76

Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping

Goyenvalle, Aurélie, Babbs, Arran, Powell, Dave, Kole, Ryszard, Fletcher, Sue, Wilton, Steve D, Davies, Kay E

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 506 KB

english, 2010

77

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

Akinc, Akin, Querbes, William, De, Soma, Qin, June, Frank-Kamenetsky, Maria, Jayaprakash, K. Narayanannair, Jayaraman, Muthusamy, Rajeev, Kallanthottathil G, Cantley, William L, Dorkin, J Robert, Butl

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 382 KB

english, 2010

78

Activation of Transgene-specific T Cells Following Lentivirus-mediated Gene Delivery to Mouse Lung

Limberis, Maria P, Bell, Christie L, Heath, Jack, Wilson, James M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 443 KB

english, 2010

79

G-rich Oligonucleotides Inhibit HIF-1α and HIF-2α and Block Tumor Growth

Guan, Yongli, Ramasamy Reddy, Kavitha, Zhu, Qiqing, Li, Yifei, Lee, KangAe, Weerasinghe, Priya, Prchal, Josef, Semenza, Gregg L, Jing, Naijie

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 815 KB

english, 2010

80

Recognition of Virus Infection and Innate Host Responses to Viral Gene Therapy Vectors

Shayakhmetov, Dmitry M, Di Paolo, Nelson C, Mossman, Karen L

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

81

Gene Therapy of Mpl Deficiency: Challenging Balance Between Leukemia and Pancytopenia

Wicke, Daniel C, Meyer, Johann, Buesche, Guntram, Heckl, Dirk, Kreipe, Hans, Li, Zhixiong, Welte, Karl H, Ballmaier, Matthias, Baum, Christopher, Modlich, Ute

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 680 KB

english, 2010

82

Efficacy and Safety/Toxicity Study of Recombinant Vaccinia Virus JX-594 in Two Immunocompetent Animal Models of Glioma

Lun, XueQing, Chan, Jennifer, Zhou, Hongyuan, Sun, Beichen, Kelly, John JP, Stechishin, Owen Owen, Bell, John C, Parato, Kelley, Hu, Kang, Vaillant, Dominique, Wang, Jiahu, Liu, Ta-Chiang, Breitbach,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 667 KB

english, 2010

83

Therapeutic Effect of Sodium Iodide Symporter Gene Therapy Combined With External Beam Radiotherapy and Targeted Drugs That Inhibit DNA Repair

Hingorani, Mohan, White, Christine L, Zaidi, Shane, Pandha, Hardev S, Melcher, Alan A, Bhide, Shreerang A, Nutting, Christopher M, Syrigos, Konstantinos N, Vile, Richard G, Vassaux, Georges, Harringto

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 389 KB

english, 2010

84

In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse

Aoki, Yoshitsugu, Nakamura, Akinori, Yokota, Toshifumi, Saito, Takashi, Okazawa, Hitoshi, Nagata, Tetsuya, Takeda, Shin'ichi

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.40 MB

english, 2010

85

Hypoxia Preconditioned Mesenchymal Stem Cells Improve Vascular and Skeletal Muscle Fiber Regeneration After Ischemia Through a Wnt4-dependent Pathway

Leroux, Lionel, Descamps, Betty, Tojais, Nancy F, Séguy, Benjamin, Oses, Pierre, Moreau, Catherine, Daret, Danièle, Ivanovic, Zoran, Boiron, Jean-Michel, Lamazière, Jean-Marie D, Dufourcq, Pascale, Co

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 801 KB

english, 2010

86

Nanoparticles Targeted With NGR Motif Deliver c-myc siRNA and Doxorubicin for Anticancer Therapy

Chen, Yunching, Wu, Jinzi J, Huang, Leaf

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 788 KB

english, 2010

87

Frequent Endonuclease Cleavage at Off-target Locations In Vivo

Petek, Lisa M, Russell, David W, Miller, Daniel G

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 186 KB

english, 2010

88

Gene Correction in Human Embryonic and Induced Pluripotent Stem Cells: Promises and Challenges Ahead

Narsinh, Kazim H, Wu, Joseph C

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 291 KB

english, 2010

89

Disrupting 5-HT2A Receptor/PDZ Protein Interactions Reduces Hyperalgesia and Enhances SSRI Efficacy in Neuropathic Pain

Pichon, Xavier, Wattiez, Anne S, Becamel, Carine, Ehrlich, Ingrid, Bockaert, Joel, Eschalier, Alain, Marin, Philippe, Courteix, Christine

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 306 KB

english, 2010

90

Metronomic Chemotherapy Enhances Antitumor Effects of Cancer Vaccine by Depleting Regulatory T Lymphocytes and Inhibiting Tumor Angiogenesis

Chen, Chi-An, Ho, Chih-Ming, Chang, Ming-Cheng, Sun, Wei-Zun, Chen, Yu-Li, Chiang, Ying-Cheng, Syu, Ming-Hong, Hsieh, Chang-Yao, Cheng, Wen-Fang

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 365 KB

english, 2010

91

Deficiency of the Housekeeping Gene Hypoxanthine–Guanine Phosphoribosyltransferase (HPRT) Dysregulates Neurogenesis

Guibinga, Ghiabe-Henri, Hsu, Stephen, Friedmann, Theodore

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

92

Evaluation of Efficacy, Biodistribution, and Inflammation for a Potent siRNA Nanoparticle: Effect of Dexamethasone Co-treatment

Abrams, Marc T, Koser, Martin L, Seitzer, Jessica, Williams, Stephanie C, DiPietro, Martha A, Wang, Weimin, Shaw, Andrew W, Mao, Xianzhi, Jadhav, Vasant, Davide, Joseph P, Burke, Paul A, Sachs, Alan B

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 273 KB

english, 2010

93

Comparative Transduction Efficiency of AAV Vector Serotypes 1–6 in the Substantia Nigra and Striatum of the Primate Brain

Markakis, Eleni A, Vives, Kenneth P, Bober, Jeremy, Leichtle, Stefan, Leranth, Csaba, Beecham, Jeff, Elsworth, John D, Roth, Robert H, Samulski, R Jude, Redmond, D Eugene

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 603 KB

english, 2010

94

Hyaluronidase Expression by an Oncolytic Adenovirus Enhances Its Intratumoral Spread and Suppresses Tumor Growth

Guedan, Sonia, Rojas, Juan José, Gros, Alena, Mercade, Elena, Cascallo, Manel, Alemany, Ramon

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 546 KB

english, 2010

95

Eight Years of Clinical Improvement in MPTP-Lesioned Primates After Gene Therapy With AAV2-hAADC

Hadaczek, Piotr, Eberling, Jamie L, Pivirotto, Philip, Bringas, John, Forsayeth, John, Bankiewicz, Krystof S

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 346 KB

english, 2010

96

A Phase I Study of Aromatic L-Amino Acid Decarboxylase Gene Therapy for Parkinson's Disease

Muramatsu, Shin-ichi, Fujimoto, Ken-ichi, Kato, Seiya, Mizukami, Hiroaki, Asari, Sayaka, Ikeguchi, Kunihiko, Kawakami, Tadataka, Urabe, Masashi, Kume, Akihiro, Sato, Toshihiko, Watanabe, Eiju, Ozawa,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 367 KB

english, 2010

97

Induction of Tumor-specific Immune Response by Gene Transfer of Hsp70-cell-penetrating Peptide Fusion Protein to Tumors in Mice

Nishikawa, Makiya, Otsuki, Takayuki, Ota, Atsushi, Guan, Xin, Takemoto, Seiji, Takahashi, Yuki, Takakura, Yoshinobu

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 281 KB

english, 2010

98

Eradication of Therapy-resistant Human Prostate Tumors Using an Ultrasound-guided Site-specific Cancer Terminator Virus Delivery Approach

Greco, Adelaide, Di Benedetto, Altomare, Howard, Candace M, Kelly, Sarah, Nande, Rounak, Dementieva, Yulia, Miranda, Michele, Brunetti, Arturo, Salvatore, Marco, Claudio, Luigi, Sarkar, Devanand, Dent

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 673 KB

english, 2010

99

Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome ≥8.2 kb

Lai, Yi, Yue, Yongping, Duan, Dongsheng

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 385 KB

english, 2010

100

Genetic-induced Variations in the GAD65 T-cell Repertoire Governs Efficacy of Anti-CD3/GAD65 Combination Therapy in New-onset Type 1 Diabetes

Bresson, Damien, Fradkin, Matthew, Manenkova, Yulia, Rottembourg, Diane, von Herrath, Matthias

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 473 KB

english, 2010

101

Multiple Functions of the 37/67-kd Laminin Receptor Make It a Suitable Target for Novel Cancer Gene Therapy

Scheiman, Jonathan, Tseng, Jen-Chieh, Zheng, Yun, Meruelo, Daniel

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 652 KB

english, 2010

102

Image-guided, Intravascular Hydrodynamic Gene Delivery to Skeletal Muscle in Pigs

Kamimura, Kenya, Zhang, Guisheng, Liu, Dexi

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 681 KB

english, 2010

103

Suppression of FVIII Inhibitor Formation in Hemophilic Mice by Delivery of Transgene Modified Apoptotic Fibroblasts

Su, Rui-Jun, Epp, Angela, Latchman, Yvette, Bolgiano, Doug, Pipe, Steven W, Josephson, Neil C

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 308 KB

english, 2010

104

Dual Reporter Comparative Indexing of rAAV Pseudotyped Vectors in Chimpanzee Airway

Flotte, Terence R, Fischer, Anne C, Goetzmann, Jason, Mueller, Christian, Cebotaru, Liudmila, Yan, Ziying, Wang, Lilli, Wilson, James M, Guggino, William B, Engelhardt, John F

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 242 KB

english, 2010

105

Enhanced Antitumor Efficacy of Vasculostatin (Vstat120) Expressing Oncolytic HSV-1

Hardcastle, Jayson, Kurozumi, Kazuhiko, Dmitrieva, Nina, Sayers, Martin P, Ahmad, Sarwat, Waterman, Peter, Weissleder, Ralph, Chiocca, E Antonio, Kaur, Balveen

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 547 KB

english, 2010

106

Polysialic Acid Glycomimetic Promotes Functional Recovery and Plasticity After Spinal Cord Injury in Mice

Mehanna, Ali, Jakovcevski, Igor, Acar, Ayşe, Xiao, Meifang, Loers, Gabriele, Rougon, Geneviève, Irintchev, Andrey, Schachner, Melitta

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.19 MB

english, 2010

107

A Self-inactivating γ-Retroviral Vector Reduces Manifestations of Mucopolysaccharidosis I in Mice

Metcalf, Jason A, Ma, Xiucui, Linders, Bruce, Wu, Susan, Schambach, Axel, Ohlemiller, Kevin K, Kovacs, Attila, Bigg, Mark, He, Li, Tollefsen, Douglas M, Ponder, Katherine P

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 392 KB

english, 2010

108

Long-term Physiologically Regulated Expression of the Low-density Lipoprotein Receptor In Vivo Using Genomic DNA Mini-gene Constructs

Hibbitt, Olivia C, McNeil, Eileen, Lufino, Michele MP, Seymour, Len, Channon, Keith, Wade-Martins, Richard

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 595 KB

english, 2010

109

BALB/c Mice Show Impaired Hepatic Tolerogenic Response Following AAV Gene Transfer to the Liver

Breous, Ekaterina, Somanathan, Suryanarayan, Wilson, James M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 394 KB

english, 2010

110

Overexpression of Sonic Hedgehog in the Lung Mimics the Effect of Lung Injury and Compensatory Lung Growth on Pulmonary Sca-1 and CD34 Positive Cells

Krause, Anja, Xu, Yaqin, Joh, Ju, Hubner, Ralf, Gess, Austen, Ilic, Thomas, Worgall, Stefan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 435 KB

english, 2010

111

Efficient KRT14 Targeting and Functional Characterization of Transplanted Human Keratinocytes for the Treatment of Epidermolysis Bullosa Simplex

Petek, Lisa M, Fleckman, Philip, Miller, Daniel G

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 698 KB

english, 2010

112

Oral Tetrahydrobiopterin Improves the Beneficial Effect of Adenoviral-mediated eNOS Gene Transfer After Induction of Hindlimb Ischemia

Yan, Jinglian, Tie, Guodong, Hoffman, Ari, Yang, Yagai, Nowicki, Philip T, Messina, Louis M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 437 KB

english, 2010

113

Aβ-directed Single-chain Antibody Delivery Via a Serotype-1 AAV Vector Improves Learning Behavior and Pathology in Alzheimer's Disease Mice

Ryan, Deborah A, Mastrangelo, Michael A, Narrow, Wade C, Sullivan, Mark A, Federoff, Howard J, Bowers, William J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 460 KB

english, 2010

114

Comparative Analysis of Immune Responses Induced by Vaccination With SIV Antigens by Recombinant Ad5 Vector or Plasmid DNA in Rhesus Macaques

Hirao, Lauren A, Wu, Ling, Satishchandran, Abhishek, Khan, Amir S, Draghia-Akli, Ruxandra, Finnefrock, Adam C, Bett, Andrew J, Betts, Michael R, Casimiro, Danilo R, Sardesai, Niranjan Y, Kim, J Joseph

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 398 KB

english, 2010

115

Real-time MR Imaging With Gadoteridol Predicts Distribution of Transgenes After Convection-enhanced Delivery of AAV2 Vectors

Su, Xiaomin, Kells, Adrian P, Aguilar Salegio, Ernesto A, Richardson, R Mark, Hadaczek, Piotr, Beyer, Janine, Bringas, John, Pivirotto, Philip, Forsayeth, John, Bankiewicz, Krystof S

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 466 KB

english, 2010

116

Ex Vivo Transduction and Transplantation of Bone Marrow Cells for Liver Gene Delivery of α1-Antitrypsin

Li, Hong, Lu, Yuanqing, Witek, Rafal P, Chang, Lung-Ji, Campbell-Thompson, Martha, Jorgensen, Marda, Petersen, Bryon, Song, Sihong

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 713 KB

english, 2010

117

Ex Vivo Expansion of Retrovirally Transduced Primate CD34+ Cells Results in Overrepresentation of Clones With MDS1/EVI1 Insertion Sites in the Myeloid Lineage After Transplantation

Sellers, Stephanie, Gomes, Theotonius J, Larochelle, Andre, Lopez, Rebecca, Adler, Rima, Krouse, Allen, Donahue, Robert E, Childs, Richard W, Dunbar, Cynthia E

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 222 KB

english, 2010

118

Exon Skipping and Duchenne Muscular Dystrophy Therapy: Selection of the Most Active U1 snRNA Antisense Able to Induce Dystrophin Exon 51 Skipping

Incitti, Tania, De Angelis, Fernanda G, Cazzella, Valentina, Sthandier, Olga, Pinnarò, Chiara, Legnini, Ivano, Bozzoni, Irene

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 292 KB

english, 2010

119

Statistical Issues in Longitudinal Data Analysis for Treatment Efficacy Studies in the Biomedical Sciences

Liu, Chunyan, Cripe, Timothy P, Kim, Mi-Ok

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 156 KB

english, 2010

120

A Ubiquitous Chromatin Opening Element (UCOE) Confers Resistance to DNA Methylation–mediated Silencing of Lentiviral Vectors

Zhang, Fang, Frost, Amy R, Blundell, Mike P, Bales, Olivia, Antoniou, Michael N, Thrasher, Adrian J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 527 KB

english, 2010

121

Urokinase-Targeted Fusion by Oncolytic Sendai Virus Eradicates Orthotopic Glioblastomas by Pronounced Synergy With Interferon-β Gene

Hasegawa, Yuzo, Kinoh, Hiroaki, Iwadate, Yasuo, Onimaru, Mitsuho, Ueda, Yasuji, Harada, Yui, Saito, Satoru, Furuya, Aki, Saegusa, Takashi, Morodomi, Yosuke, Hasegawa, Mamoru, Saito, Shigeyoshi, Aoki,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 358 KB

english, 2010

122

A Therapeutic Strategy for Choroidal Neovascularization Based on Recruitment of Mesenchymal Stem Cells to the Sites of Lesions

Hou, Hui-Yuan, Liang, Hong-Liang, Wang, Yu-Sheng, Zhang, Zhao-Xia, Wang, Bai-Ren, Shi, Yuan-Yuan, Dong, Xiao, Cai, Yan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 907 KB

english, 2010

123

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

Yin, HaiFang, Moulton, Hong M, Betts, Corinne, Merritt, Thomas, Seow, Yiqi, Ashraf, Shirin, Wang, QingSong, Boutilier, Jordan, Wood, Matthew JA

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 852 KB

english, 2010

124

2010 Gene Therapy Year in Review

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 193 KB

english, 2010

125

Chemotherapy Delivered After Viral Immunogene Therapy Augments Antitumor Efficacy Via Multiple Immune-mediated Mechanisms

Fridlender, Zvi G, Sun, Jing, Singhal, Sunil, Kapoor, Veena, Cheng, Guanjun, Suzuki, Eiji, Albelda, Steven M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 443 KB

english, 2010

126

Is It Safer CARs That We Need, or Safer Rules of the Road?

Junghans, Richard P

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 59 KB

english, 2010

127

Selectivity of Oncolytic Viral Replication Prevents Antiviral Immune Response and Toxicity, but Does Not Improve Antitumoral Immunity

Gürlevik, Engin, Woller, Norman, Strüver, Nina, Schache, Peter, Kloos, Arnold, Manns, Michael P, Zender, Lars, Kühnel, Florian, Kubicka, Stefan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 621 KB

english, 2010

128

Laminin-111: A Potential Therapeutic Agent for Duchenne Muscular Dystrophy

Goudenege, Sébastien, Lamarre, Yann, Dumont, Nicolas, Rousseau, Joël, Frenette, Jérôme, Skuk, Daniel, Tremblay, Jacques P

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 463 KB

english, 2010

129

High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines

Markusic, David M, Herzog, Roland W, Aslanidi, George V, Hoffman, Brad E, Li, Baozheng, Li, Mengxin, Jayandharan, Giridhara R., Ling, Chen, Zolotukhin, Irene, Ma, Wenqin, Zolotukhin, Sergei, Srivastav

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 668 KB

english, 2010

130

Minimal RB-responsive E1A Promoter Modification to Attain Potency, Selectivity, and Transgene-arming Capacity in Oncolytic Adenoviruses

Rojas, Juan J, Guedan, Sonia, Searle, Peter F, Martinez-Quintanilla, Jordi, Gil-Hoyos, Raúl, Alcayaga-Miranda, Francisca, Cascallo, Manel, Alemany, Ramon

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 540 KB

english, 2010

131

Helper-dependent Adenoviral Vectors are Superior In Vitro to First-generation Vectors for Endothelial Cell-targeted Gene Therapy

Flynn, Rowan, Buckler, Joshua M, Tang, Chongren, Kim, Francis, Dichek, David A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 420 KB

english, 2010

132

Gene Therapy and Bone Marrow Transplantation for Thalassemia: Changing of the Guard?

Walters, Mark C

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 135 KB

english, 2010

133

Prophylactic Antiparasitic Transgenesis for Human Parasitic Disease?

Lukeš, Julius, Raper, Jayne

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 421 KB

english, 2010

134

Retraction to”Metabolic Basis of Sexual Dimorphism in PKU Mice After Genome-targeted PAH Gene Therapy”

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 883 KB

english, 2010

135

AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice

Dodge, James C, Treleaven, Christopher M, Fidler, Jonathan A, Hester, Mark, Haidet, Amanda, Handy, Chalonda, Rao, Meghan, Eagle, Amy, Matthews, Jennifer C, Taksir, Tatyana V, Cheng, Seng H, Shihabuddi

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 741 KB

english, 2010

136

A Fusion of GMCSF and IL-21 Initiates Hypersignaling Through the IL-21Rα Chain With Immune Activating and Tumoricidal Effects In Vivo

Williams, Patrick, Rafei, Moutih, Bouchentouf, Manaf, Raven, Jennifer, Yuan, Shala, Cuerquis, Jessica, Forner, Kathy A, Birman, Elena, Galipeau, Jacques

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 343 KB

english, 2010

137

Complete Normalization of Hepatic G6PC Deficiency in Murine Glycogen Storage Disease Type Ia Using Gene Therapy

Yiu, Wai Han, Lee, Young Mok, Peng, Wen-Tao, Pan, Chi-Jiunn, Mead, Paul A, Mansfield, Brian C, Chou, Janice Y

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 472 KB

english, 2010

138

Vasoactive Intestinal Peptide Increases Hepatic Transduction and Reduces Innate Immune Response Following Administration of Helper-dependent Ad

Vetrini, Francesco, Brunetti-Pierri, Nicola, Palmer, Donna J, Bertin, Terry, Grove, Nathan C, Finegold, Milton J, Ng, Philip

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

139

Abstracts

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 12.55 MB

english, 2010

140

SIN Retroviral Vectors Expressing COL7A1 Under Human Promoters for Ex Vivo Gene Therapy of Recessive Dystrophic Epidermolysis Bullosa

Titeux, Matthias, Pendaries, Valérie, Zanta-Boussif, Maria A, Décha, Audrey, Pironon, Nathalie, Tonasso, Laure, Mejia, José E, Brice, Agnes, Danos, Olivier, Hovnanian, Alain

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 586 KB

english, 2010

141

Transient Demyelination Increases the Efficiency of Retrograde AAV Transduction

Hollis, Edmund R, Jamshidi, Pouya, Lorenzana, Ariana O, Lee, Jae K, Gray, Steven J, Samulski, Richard J, Zheng, Binhai, Tuszynski, Mark H

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 306 KB

english, 2010

142

An Improved Bicistronic CD20/tCD34 Vector for Efficient Purification and In Vivo Depletion of Gene-Modified T Cells for Adoptive Immunotherapy

Vogler, Isabel, Newrzela, Sebastian, Hartmann, Sylvia, Schneider, Nadine, von Laer, Dorothee, Koehl, Ulrike, Grez, Manuel

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 344 KB

english, 2010

143

Cellular Antioxidant Levels Influence Muscle Stem Cell Therapy

Drowley, Lauren, Okada, Masaho, Beckman, Sarah, Vella, Joseph, Keller, Bradley, Tobita, Kimimasa, Huard, Johnny

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 446 KB

english, 2010

144

Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models

Wang, Lili, Wang, Huan, Bell, Peter, McCarter, Robert J, He, Jianping, Calcedo, Roberto, Vandenberghe, Luk H, Morizono, Hiroki, Batshaw, Mark L, Wilson, James M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 490 KB

english, 2010

145

Amphiregulin Promotes BAX Inhibition and Resistance to Gefitinib in Non-small-cell Lung Cancers

Busser, Benoît, Sancey, Lucie, Josserand, Véronique, Niang, Carole, Favrot, Marie C, Coll, Jean-Luc, Hurbin, Amandine

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 300 KB

english, 2010

146

Intensive Pharmacological Immunosuppression Allows for Repetitive Liver Gene Transfer With Recombinant Adenovirus in Nonhuman Primates

Fontanellas, Antonio, Hervás-Stubbs, Sandra, Mauleón, Itsaso, Dubrot, Juan, Mancheño, Uxua, Collantes, María, Sampedro, Ana, Unzu, Carmen, Alfaro, Carlos, Palazón, Asis, Smerdou, Cristian, Benito, Alb

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 1.15 MB

2010

147

HIV Sequence Variation Associated With env Antisense Adoptive T-cell Therapy in the hNSG Mouse Model

Mukherjee, Rithun, Plesa, Gabriela, Sherrill-Mix, Scott, Richardson, Max W, Riley, James L, Bushman, Frederic D

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 364 KB

2010

148

Treatment of Cancer Patients With a Serotype 5/3 Chimeric Oncolytic Adenovirus Expressing GMCSF

Koski, Anniina, Kangasniemi, Lotta, Escutenaire, Sophie, Pesonen, Sari, Cerullo, Vincenzo, Diaconu, Iulia, Nokisalmi, Petri, Raki, Mari, Rajecki, Maria, Guse, Kilian, Ranki, Tuuli, Oksanen, Minna, Hol

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 324 KB

2010

149

Screening of Complement Inhibitors: Shielded Baculoviruses Increase the Safety and Efficacy of Gene Delivery

Kaikkonen, Minna U, Maatta, Antti I, Ylä-Herttuala, Seppo, Airenne, Kari J

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 256 KB

2010

150

SERCA2a Gene Transfer Enhances eNOS Expression and Activity in Endothelial Cells

Hadri, Lahouaria, Bobe, Regis, Kawase, Yoshiaki, Ladage, Dennis, Ishikawa, Kiyotake, Atassi, Fabrice, Lebeche, Djamel, Kranias, Evangelia G, Leopold, Jane A, Lompré, Anne-Marie, Lipskaia, Larissa, Haj

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 571 KB

2010

151

Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease

Sun, Baodong, Kulis, Michael D, Young, Sarah P, Hobeika, Amy C, Li, Songtao, Bird, Andrew, Zhang, Haoyue, Li, Yifan, Clay, Timothy M, Burks, Wesley, Kishnani, Priya S, Koeberl, Dwight D

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 273 KB

english, 2010

152

Low-level shRNA Cytotoxicity Can Contribute to MYC-induced Hepatocellular Carcinoma in Adult Mice

Beer, Shelly, Bellovin, David I, Lee, Joyce S, Komatsubara, Kimberly, Wang, Lora S, Koh, Huishan, Börner, Kathleen, Storm, Theresa A, Davis, Corrine R, Kay, Mark A, Felsher, Dean W, Grimm, Dirk

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 730 KB

english, 2010

153

Amphiregulin Promotes Resistance to Gefitinib in NonSmall Cell Lung Cancer Cells by Regulating Ku70 Acetylation

Busser, Benoît, Sancey, Lucie, Josserand, Véronique, Niang, Carole, Khochbin, Saadi, Favrot, Marie C, Coll, Jean-Luc, Hurbin, Amandine

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 356 KB

english, 2010

154

The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques

Wang, Lili, Calcedo, Roberto, Wang, Huan, Bell, Peter, Grant, Rebecca, Vandenberghe, Luk H, Sanmiguel, Julio, Morizono, Hiroki, Batshaw, Mark L, Wilson, James M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 388 KB

english, 2010

155

Pretranscriptional Regulation of Tgf-β1 by PI Polyamide Prevents Scarring and Accelerates Wound Healing of the Cornea After Exposure to Alkali

Chen, Min, Matsuda, Hiroyuki, Wang, Linghua, Watanabe, Takayoshi, Kimura, Makoto T, Igarashi, Jun, Wang, Xiaofei, Sakimoto, Tohru, Fukuda, Noboru, Sawa, Mitsuru, Nagase, Hiroki

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 699 KB

english, 2010

156

Little Vector, Big Gene Transduction: Fragmented Genome Reassembly of Adeno-associated Virus

Hirsch, Matthew L, Agbandje-McKenna, Mavis, Jude Samulski, R

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 283 KB

english, 2010

157

Cocal-pseudotyped Lentiviral Vectors Resist Inactivation by Human Serum and Efficiently Transduce Primate Hematopoietic Repopulating Cells

Trobridge, Grant D, Wu, Robert A, Hansen, Michael, Ironside, Christina, Watts, Korashon L, Olsen, Philip, Beard, Brian C, Kiem, Hans-Peter

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

158

Adoptive Transfer of Ex Vivo HO-1 Modified Bone Marrow–derived Macrophages Prevents Liver Ischemia and Reperfusion Injury

Ke, Bibo, Shen, Xiu-Da, Gao, Feng, Ji, Haofeng, Qiao, Bo, Zhai, Yuan, Farmer, Douglas G, Busuttil, Ronald W, Kupiec-Weglinski, Jerzy W

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 571 KB

english, 2010

159

Dendritic Cells Transduced With Lentiviral Vectors Expressing VIP Differentiate Into VIP-secreting Tolerogenic-like DCs

Toscano, Miguel G, Delgado, Mario, Kong, Weimin, Martin, Francisco, Skarica, Mario, Ganea, Doina

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 463 KB

english, 2010

160

Immune Responses to AAV in Canine Muscle Monitored by Cellular Assays and Noninvasive Imaging

Wang, Zejing, Storb, Rainer, Lee, Donghoon, Kushmerick, Martin J, Chu, Baocheng, Berger, Carolina, Arnett, Andrea, Allen, James, Chamberlain, Jeffrey S, Riddell, Stanley R, Tapscott, Stephen J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 819 KB

english, 2010

161

Zinc-finger Nuclease-induced Gene Repair With Oligodeoxynucleotides: Wanted and Unwanted Target Locus Modifications

Radecke, Sarah, Radecke, Frank, Cathomen, Toni, Schwarz, Klaus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 433 KB

english, 2010

162

A Phase I Trial of Repeated Intrapleural Adenoviral-mediated Interferon-β Gene Transfer for Mesothelioma and Metastatic Pleural Effusions

Sterman, Daniel H, Recio, Adri, Haas, Andrew R, Vachani, Anil, Katz, Sharyn I, Gillespie, Colin T, Cheng, Guanjun, Sun, Jing, Moon, Edmund, Pereira, Luana, Wang, Xinzhong, Heitjan, Daniel F, Litzky, L

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 354 KB

english, 2010

163

Combination of Myostatin Pathway Interference and Dystrophin Rescue Enhances Tetanic and Specific Force in Dystrophic mdx Mice

Dumonceaux, Julie, Marie, Solenne, Beley, Cyriaque, Trollet, Capucine, Vignaud, Alban, Ferry, Arnaud, Butler-Browne, Gillian, Garcia, Luis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 282 KB

english, 2010

164

TLR9 and IRF3 Cooperate to Induce a Systemic Inflammatory Response in Mice Injected With Liposome:DNA

Walker, Wendy E, Booth, Carmen J, Goldstein, Daniel R

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 396 KB

english, 2010

165

The Birth of the American Society of Gene Therapy

Stamatoyannopoulos, George

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 212 KB

english, 2010

166

Introduction of an N-Glycan Sequon Into HEXA Enhances Human β-Hexosaminidase Cellular Uptake in a Model of Sandhoff Disease

Matsuoka, Kazuhiko, Tsuji, Daisuke, Aikawa, Sei-ichi, Matsuzawa, Fumiko, Sakuraba, Hitoshi, Itoh, Kohji

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 439 KB

english, 2010

167

The Eyes Have It

Brenner, Malcolm

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 202 KB

english, 2010

168

Robust In Vivo Transduction of Nervous System and Neural Stem Cells by Early Gestational Intra Amniotic Gene Transfer Using Lentiviral Vector

Stitelman, David H, Endo, Masayuki, Bora, Archana, Muvarak, Nidal, Zoltick, Philip W, Flake, Alan W, Brazelton, Timothy R

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.91 MB

english, 2010

169

Absence of Systemic Immune Response to Adenovectors After Intraocular Administration to Children With Retinoblastoma

Ildefonso, Cristhian J, Kong, Lingkun, Leen, Ann, Chai, Samantha J, Petrochelli, Veronica, Chintagumpala, Murali, Hurwitz, Mary Y, Chévez-Barrios, Patricia, Hurwitz, Richard L

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

170

Induction of Immune Tolerance to a Therapeutic Protein by Intrathymic Gene Delivery

Chu, Qiuming, Moreland, Rodney J, Gao, Lan, Taylor, Kristin M, Meyers, Elizabeth, Cheng, Seng H, Scheule, Ronald K

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 330 KB

english, 2010

171

Breathing New Life Into Lung Transplantation Therapy

Panoskaltsis-Mortari, Angela, Weiss, Daniel J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 537 KB

english, 2010

172

Nodal/Activin Signaling Predicts Human Pluripotent Stem Cell Lines Prone to Differentiate Toward the Hematopoietic Lineage

Ramos-Mejia, Veronica, Melen, Gustavo J, Sanchez, Laura, Gutierrez-Aranda, Ivan, Ligero, Gertrudis, Cortes, Jose L, Real, Pedro J, Bueno, Clara, Menendez, Pablo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 596 KB

english, 2010

173

The Effect of Cell Cycle Synchronization on Tumor Sensitivity to Reovirus Oncolysis

Heinemann, Lucy, Simpson, Guy R, Annels, Nicola E, Vile, Richard, Melcher, Alan, Prestwich, Robin, Harrington, Kevin J, Pandha, Hardev S

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 365 KB

english, 2010

174

Comparison of AAV Serotypes for Gene Delivery to Dorsal Root Ganglion Neurons

Mason, Matthew RJ, Ehlert, Erich ME, Eggers, Ruben, Pool, Chris W, Hermening, Stephan, Huseinovic, Angelina, Timmermans, Eric, Blits, Bas, Verhaagen, Joost

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.63 MB

english, 2010

175

Expansive Gene Transfer in the Rat CNS Rapidly Produces Amyotrophic Lateral Sclerosis Relevant Sequelae When TDP-43 is Overexpressed

Wang, David B, Dayton, Robert D, Henning, Phillip P, Cain, Cooper D, Zhao, Li Ru, Schrott, Lisa M, Orchard, Elysse A, Knight, David S, Klein, Ronald L

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.33 MB

english, 2010

176

Clinical Success of Complex Biological Therapies: Be Careful What You Wish For...

Brenner, Malcolm K

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 182 KB

english, 2010

177

Transatlantic Consortium Spotlights Need for Changes in Gene Therapy Trials

Williams, David A, Thrasher, Adrian J, Baum, Christopher

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 118 KB

english, 2010

178

The Need for Genetically Engineering Therapeutic Pluripotent Stem Cells

Sadelain, Michel

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 115 KB

english, 2010

179

The Stem Cell “Tunnel” Effect

Zwaka, Thomas P

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 201 KB

english, 2010

180

Processing of Tumor Antigen Differentially Impacts the Development of Helper and Effector CD4+ T-cell Responses

Bernard, Dannie, Ventresca, Michael S, Marshall, Laura A, Evelegh, Carole, Wan, Yonghong, Bramson, Jonathan L

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 250 KB

english, 2010

181

LEDGF Hybrids Efficiently Retarget Lentiviral Integration Into Heterochromatin

Gijsbers, Rik, Ronen, Keshet, Vets, Sofie, Malani, Nirav, De Rijck, Jan, McNeely, Melissa, Bushman, Frederic D, Debyser, Zeger

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 367 KB

english, 2010

182

Systemic Therapy of Disseminated Myeloma in Passively Immunized Mice Using Measles Virus-infected Cell Carriers

Liu, Chunsheng, Russell, Stephen J, Peng, Kah-Whye

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.15 MB

english, 2010

183

Phase II Clinical Trial of Intratumoral Application of TG1042 (Adenovirus-interferon-γ) in Patients With Advanced Cutaneous T-cell Lymphomas and Multilesional Cutaneous B-cell Lymphomas

Dummer, Reinhard, Eichmüller, Stefan, Gellrich, Sylke, Assaf, Chalid, Dreno, Brigitte, Schiller, Meinhard, Dereure, Olivier, Baudard, Marion, Bagot, Martine, Khammari, Amir, Bleuzen, Pascal, Bataille,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 166 KB

english, 2010

184

Lentiviral Vector Gene Therapy: Effective and Safe?

Persons, Derek A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 211 KB

english, 2010

185

FimH Can Directly Activate Human and Murine Natural Killer Cells via TLR4

Mian, M Firoz, Lauzon, Nicole M, Andrews, David W, Lichty, Brian D, Ashkar, Ali A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 447 KB

english, 2010

186

Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs

Kornegay, Joe N, Li, Juan, Bogan, Janet R, Bogan, Daniel J, Chen, Chunlian, Zheng, Hui, Wang, Bing, Qiao, Chunping, Howard, James F, Xiao, Xiao

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.14 MB

english, 2010

187

B-Cell-Delivered Gene Therapy Induces Functional T Regulatory Cells and Leads to a Loss of Antigen-Specific Effector Cells

Skupsky, Jonathan, Zhang, Ai-Hong, Su, Yan, Scott, David W

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 352 KB

english, 2010

188

Adeno-associated Virus Gene Therapy With Cholesterol 24-Hydroxylase Reduces the Amyloid Pathology Before or After the Onset of Amyloid Plaques in Mouse Models of Alzheimer's Disease

Hudry, Eloise, Van Dam, Debby, Kulik, Wim, De Deyn, Peter P, Stet, Femke S, Ahouansou, Ornella, Benraiss, Abdellatif, Delacourte, André, Bougnères, Pierre, Aubourg, Patrick, Cartier, Nathalie

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 583 KB

english, 2010

189

Regulatory T Cells and Immune Tolerance to Coagulation Factor IX in the Context of Intramuscular AAV1 Gene Transfer

Kelly, Meagan, Bharadwaj, Arpita S, Tacke, Frank, Chao, Hengjun

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 451 KB

english, 2010

190

Enhancing the Therapeutic Effect Against Ovarian Cancer Through a Combination of Viral Oncolysis and Antigen-specific Immunotherapy

Zhang, Yu-Qian, Tsai, Ya-Chea, Monie, Archana, Wu, T.-C., Hung, Chien-Fu

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 638 KB

english, 2010

191

Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy

Hurlbut, Gregory D, Ziegler, Robin J, Nietupski, Jennifer B, Foley, Joseph W, Woodworth, Lisa A, Meyers, Elizabeth, Bercury, Scott D, Pande, Nilesh N, Souza, David W, Bree, Mark P, Lukason, Michael J,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 321 KB

english, 2010

192

Gene Therapy for SCID-X1: Round 2

Herzog, Roland W

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 122 KB

english, 2010

193

Intensive Pharmacological Immunosuppression Allows for Repetitive Liver Gene Transfer With Recombinant Adenovirus in Nonhuman Primates

Fontanellas, Antonio, Hervás-Stubbs, Sandra, Mauleón, Itsaso, Dubrot, Juan, Mancheño, Uxua, Collantes, María, Sampedro, Ana, Unzu, Carmen, Alfaro, Carlos, Palazón, Asis, Smerdou, Cristian, Benito, Alb

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.15 MB

english, 2010

194

HIV Sequence Variation Associated With env Antisense Adoptive T-cell Therapy in the hNSG Mouse Model

Mukherjee, Rithun, Plesa, Gabriela, Sherrill-Mix, Scott, Richardson, Max W, Riley, James L, Bushman, Frederic D

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 364 KB

english, 2010

195

Treatment of Cancer Patients With a Serotype 5/3 Chimeric Oncolytic Adenovirus Expressing GMCSF

Koski, Anniina, Kangasniemi, Lotta, Escutenaire, Sophie, Pesonen, Sari, Cerullo, Vincenzo, Diaconu, Iulia, Nokisalmi, Petri, Raki, Mari, Rajecki, Maria, Guse, Kilian, Ranki, Tuuli, Oksanen, Minna, Hol

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 324 KB

english, 2010

196

Screening of Complement Inhibitors: Shielded Baculoviruses Increase the Safety and Efficacy of Gene Delivery

Kaikkonen, Minna U, Maatta, Antti I, Ylä-Herttuala, Seppo, Airenne, Kari J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

197

SERCA2a Gene Transfer Enhances eNOS Expression and Activity in Endothelial Cells

Hadri, Lahouaria, Bobe, Regis, Kawase, Yoshiaki, Ladage, Dennis, Ishikawa, Kiyotake, Atassi, Fabrice, Lebeche, Djamel, Kranias, Evangelia G, Leopold, Jane A, Lompré, Anne-Marie, Lipskaia, Larissa, Haj

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 571 KB

english, 2010

198

Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction

Finn, Jonathan D, Hui, Daniel, Downey, Harre D, Dunn, Danielle, Pien, Gary C, Mingozzi, Federico, Zhou, Shangzhen, High, Katherine A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 261 KB

english, 2010

199

Stem Cell Vaccination Against Cancer: Fighting Fire With Fire?

Zwaka, Thomas P

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 282 KB

english, 2010

200

Transplantation of Human Embryonic Stem Cell–Derived Alveolar Epithelial Type II Cells Abrogates Acute Lung Injury in Mice

Wang, Dachun, Morales, John E, Calame, Daniel G, Alcorn, Joseph L, Wetsel, Rick A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 781 KB

english, 2010

201

Efficient Gene Transfer Into the Mouse Lung by Fetal Intratracheal Injection of rAAV2/6.2

Carlon, Marianne, Toelen, Jaan, Van der Perren, Anke, Vandenberghe, Luk H, Reumers, Veerle, Sbragia, Lourenço, Gijsbers, Rik, Baekelandt, Veerle, Himmelreich, Uwe, Wilson, James M, Deprest, Jan, Debys

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 787 KB

english, 2010

202

Regulation of Retinal Function but Nonrescue of Vision in RPE65-deficient Dogs Treated With Doxycycline-regulatable AAV Vectors

Lhériteau, Elsa, Libeau, Lyse, Mendes-Madeira, Alexandra, Deschamps, Jack-Yves, Weber, Michel, Le Meur, Guylène, Provost, Nathalie, Guihal, Caroline, Moullier, Philippe, Rolling, Fabienne

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 336 KB

english, 2010

203

Embryonic Stem Cells and Repair of Lung Injury

Weiss, Daniel J, Finck, Christine

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 287 KB

english, 2010

204

Prevention of Senescence Progression in Reversibly Immortalized Human Ensheathing Glia Permits Their Survival After Deimmortalization

García-Escudero, Vega, García-Gómez, Ana, Gargini, Ricardo, Martín-Bermejo, María J, Langa, Elena, de Yébenes, Justo G, Delicado, Alicia, Ávila, Jesús, Moreno-Flores, María T, Lim, Filip

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 459 KB

english, 2010

205

Current Issues and Future Directions of Oncolytic Adenoviruses

Yamamoto, Masato, Curiel, David T

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 275 KB

english, 2010

206

Of Mice, Not Men: No Evidence for Graft-versus-Host Disease in Humans Receiving T-Cell Receptor–Transduced Autologous T Cells

Rosenberg, Steven A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 231 KB

english, 2010

207

Transduction of Human Primitive Repopulating Hematopoietic Cells With Lentiviral Vectors Pseudotyped With Various Envelope Proteins

Kim, Yoon-Sang, Wielgosz, Matthew M, Hargrove, Phillip, Kepes, Steven, Gray, John, Persons, Derek A, Nienhuis, Arthur W

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 250 KB

english, 2010

208

The Extracellular Matrix Glycoprotein Tenascin-C Is Beneficial for Spinal Cord Regeneration

Chen, Jian, Joon Lee, Hyun, Jakovcevski, Igor, Shah, Ronak, Bhagat, Neha, Loers, Gabriele, Liu, Hsing-Yin, Meiners, Sally, Taschenberger, Grit, Kügler, Sebastian, Irintchev, Andrey, Schachner, Melitta

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 507 KB

english, 2010

209

Restoration of Cone Vision in the CNGA3−/− Mouse Model of Congenital Complete Lack of Cone Photoreceptor Function

Michalakis, Stylianos, Mühlfriedel, Regine, Tanimoto, Naoyuki, Krishnamoorthy, Vidhyasankar, Koch, Susanne, Fischer, M Dominik, Becirovic, Elvir, Bai, Lin, Huber, Gesine, Beck, Susanne C, Fahl, Edda,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 591 KB

english, 2010

210

Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)

Gray, Steven J, Blake, Bonita L, Criswell, Hugh E, Nicolson, Sarah C, Samulski, R Jude, McCown, Thomas J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 733 KB

english, 2010

211

Silencing of Reporter Gene Expression in Skin Using siRNAs and Expression of Plasmid DNA Delivered by a Soluble Protrusion Array Device (PAD)

Gonzalez-Gonzalez, Emilio, Speaker, Tycho J, Hickerson, Robyn P, Spitler, Ryan, Flores, Manuel A, Leake, Devin, Contag, Christopher H, Kaspar, Roger L

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 773 KB

english, 2010

212

Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle

Toromanoff, Alice, Adjali, Oumeya, Larcher, Thibaut, Hill, Marcelo, Guigand, Lydie, Chenuaud, Pierre, Deschamps, Jack-Yves, Gauthier, Olivier, Blancho, Gilles, Vanhove, Bernard, Rolling, Fabienne, Ché

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 513 KB

english, 2010

213

Modulation of Exaggerated-IgE Allergic Responses by Gene Transfer-mediated Antagonism of IL-13 and IL-17e

Mueller, Christian, Keeler, Allison, Braag, Sofia, Menz, Timothy, Tang, Qiushi, Flotte, Terence R

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

214

Enhanced Antitumor Effects of an Engineered Measles Virus Edmonston Strain Expressing the Wild-type N, P, L Genes on Human Renal Cell Carcinoma

Meng, Xin, Nakamura, Takafumi, Okazaki, Toshihiko, Inoue, Hiroyuki, Takahashi, Atsushi, Miyamoto, Shohei, Sakaguchi, Gaku, Eto, Masatoshi, Naito, Seiji, Takeda, Makoto, Yanagi, Yusuke, Tani, Kenzaburo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 270 KB

english, 2010

215

Guanine Analogues Enhance Antisense Oligonucleotide-induced Exon Skipping in Dystrophin Gene In Vitro and In Vivo

Hu, Yihong, Wu, Bo, Zillmer, Allen, Lu, Peijuan, Benrashid, Ehsan, Wang, Mingxing, Doran, Timothy, Shaban, Mona, Wu, Xiaohua, Long Lu, Qi

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 576 KB

english, 2010

216

Designer Lipids Advance Systemic siRNA Delivery

Liu, Yang, Huang, Leaf

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 253 KB

english, 2010

217

In Vivo Delivery of Antigens by Adenovirus Dodecahedron Induces Cellular and Humoral Immune Responses to Elicit Antitumor Immunity

Villegas-Mendez, Ana, Garin, Marina I, Pineda-Molina, Estela, Veratti, Eugenia, Bueren, Juan A, Fender, Pascal, Lenormand, Jean-Luc

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 389 KB

english, 2010

218

Liver Regeneration From Induced Pluripotent Stem Cells

Cheng, Xin, Gadue, Paul

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

219

Macrophages Expressing Heme Oxygenase-1 Improve Renal Function in Ischemia/Reperfusion Injury

Ferenbach, David A, Ramdas, Vasudev, Spencer, Nishrin, Marson, Lorna, Anegon, Ignacio, Hughes, Jeremy, Kluth, David C

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 615 KB

english, 2010

220

Biosafety Assessment of Site-directed Transgene Integration in Human Umbilical Cord–lining Cells

Sivalingam, Jaichandran, Krishnan, Shruti, Ng, Wai Har, Lee, Sze Sing, Phan, Toan Thang, Kon, Oi Lian

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 652 KB

english, 2010

221

Regulated Expansion of Human Pancreatic β-Cells

Pais, Eszter, Park, Jean, Alexy, Tamas, Nikolian, Vahagn, Ge, Shundi, Shaw, Kit, Senadheera, Shantha, Hardee, Cinnamon L, Skelton, Dianne, Hollis, Roger, Crooks, Gay M, Kohn, Donald B

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 508 KB

english, 2010

222

Hepatic AAV Gene Transfer and the Immune System: Friends or Foes?

Herzog, Roland W

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 362 KB

english, 2010

223

Low Oxygen Tension and Synthetic Nanogratings Improve the Uniformity and Stemness of Human Mesenchymal Stem Cell Layer

Zhao, Feng, Veldhuis, Jan J, Duan, Yajun, Yang, Yong, Christoforou, Nicolas, Ma, Teng, Leong, Kam W

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.00 MB

english, 2010

224

Differential Transduction Following Basal Ganglia Administration of Distinct Pseudotyped AAV Capsid Serotypes in Nonhuman Primates

Dodiya, Hemraj B, Bjorklund, Tomas, Stansell III, James, Mandel, Ronald J, Kirik, Deniz, Kordower, Jeffrey H

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.33 MB

english, 2010

225

Safer CARS

Heslop, Helen E

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

226

DYS-HAC-iPS Cells: The Combination of Gene and Cell Therapy to Treat Duchenne Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 364 KB

english, 2010

227

Autoimmunity, Recessive Diseases, and Gene Replacement Therapy

Wilson, James M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 203 KB

english, 2010

228

Enhanced Induction of HIV-specific Cytotoxic T Lymphocytes by Dendritic Cell-targeted Delivery of SOCS-1 siRNA

Subramanya, Sandesh, Armant, Myriam, Salkowitz, Janelle R, Nyakeriga, Alice M, Haridas, Viraga, Hasan, Maroof, Bansal, Anju, Goepfert, Paul A, Wynn, Katherine K, Ladell, Kristin, Price, David A, N, Ma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 427 KB

english, 2010

229

Long-term Delivery of Nerve Growth Factor by Encapsulated Cell Biodelivery in the Göttingen Minipig Basal Forebrain

Fjord-Larsen, Lone, Kusk, Philip, Tornøe, Jens, Juliusson, Bengt, Torp, Malene, Bjarkam, Carsten R, Nielsen, Mette S, Handberg, Aase, Sørensen, Jens Christian H, Wahlberg, Lars U

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 613 KB

english, 2010

230

Therapeutic Efficacy of Bone Marrow Transplant, Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB

Heldermon, Coy D, Ohlemiller, Kevin K, Herzog, Erik D, Vogler, Carole, Qin, Elizabeth, Wozniak, David F, Tan, Yun, Orrock, John L, Sands, Mark S

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 383 KB

english, 2010

231

Ad.Egr-TNF and Local Ionizing Radiation Suppress Metastases by Interferon-β-Dependent Activation of Antigen-specific CD8+ T Cells

Meng, Yuru, Mauceri, Helena J, Khodarev, Nikolai N, Darga, Thomas E, Pitroda, Sean P, Beckett, Michael A, Kufe, Donald W, Weichselbaum, Ralph R

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 595 KB

english, 2010

232

Oncolytic Viruses: Now Interviewing for the All-Star Game

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 280 KB

english, 2010

233

Guanidinylated Neomycin Mediates Heparan Sulfate–dependent Transport of Active Enzymes to Lysosomes

Sarrazin, Stéphane, Wilson, Beth, Sly, William S, Tor, Yitzhak, Esko, Jeffrey D

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 280 KB

english, 2010

234

Rescue of a Dystrophin-like Protein by Exon Skipping In Vivo Restores GABAA-receptor Clustering in the Hippocampus of the mdx Mouse

Vaillend, Cyrille, Perronnet, Caroline, Ros, Carine, Gruszczynski, Carole, Goyenvalle, Aurélie, Laroche, Serge, Danos, Olivier, Garcia, Luis, Peltekian, Elise

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 457 KB

english, 2010

235

T Cells Expressing Constitutively Active Akt Resist Multiple Tumor-associated Inhibitory Mechanisms

Sun, Jiali, Dotti, Gianpietro, Huye, Leslie E, Foster, Aaron E, Savoldo, Barbara, Gramatges, Maria M, Spencer, David M, Rooney, Cliona M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 615 KB

english, 2010

236

A Universal Influenza A Vaccine Based on Adenovirus Expressing Matrix-2 Ectodomain and Nucleoprotein Protects Mice From Lethal Challenge

Zhou, Dongming, Wu, Te-Lang, Lasaro, Marcio O, Latimer, Brian P, Parzych, Elizabeth M, Bian, Ang, Li, Yan, Li, Hua, Erikson, Jan, Xiang, Zhiquan, Ertl, Hildegund CJ

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 395 KB

english, 2010

237

Detrimental Effect of the Proteasome Inhibitor, Bortezomib in Bacterial Superantigen- and Lipopolysaccharide-induced Systemic Inflammation

Tilahun, Ashenafi Y, Theuer, Jayne E, Patel, Robin, David, Chella S, Rajagopalan, Govindarajan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 727 KB

english, 2010

238

Assessing the Risk of T-cell Malignancies in Mouse Models of SCID-X1

Sorrentino, Brian

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 265 KB

english, 2010

239

Successful Treatment of Metachromatic Leukodystrophy Using Bone Marrow Transplantation of HoxB4 Overexpressing Cells

Miyake, Noriko, Miyake, Koichi, Karlsson, Stefan, Shimada, Takashi

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 461 KB

english, 2010

240

Desmin-regulated Lentiviral Vectors for Skeletal Muscle Gene Transfer

Talbot, Gillian E, Waddington, Simon N, Bales, Olivia, Tchen, Rose C, Antoniou, Michael N

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 286 KB

english, 2010

241

Prostaglandin Pathway Gene Therapy for Sustained Reduction of Intraocular Pressure

Barraza, Román A, McLaren, Jay W, Poeschla, Eric M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 556 KB

english, 2010

242

Reducible Poly(oligo-D-arginine) for Enhanced Gene Expression in Mouse Lung by Intratracheal Injection

Won, Young-Wook, Kim, Hyun Ah, Lee, Minhyung, Kim, Yong-Hee

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 674 KB

english, 2010

243

Potent Oncolytic Activity of Raccoonpox Virus in the Absence of Natural Pathogenicity

Evgin, Laura, Vähä-Koskela, Markus, Rintoul, Julia, Falls, Theresa, Le Boeuf, Fabrice, Barrett, John W, Bell, John C, Stanford, Marianne M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 288 KB

english, 2010

244

Transcriptional and Translational Dual-regulated Oncolytic Herpes Simplex Virus Type 1 for Targeting Prostate Tumors

Lee, Cleo YF, Bu, Luke XX, DeBenedetti, Arrigo, Williams, B Jill, Rennie, Paul S, Jia, William WG

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

245

Hybrid Lentiviral Vectors

Qasim, Waseem, Vink, Conrad A, Thrasher, Adrian J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 149 KB

english, 2010

246

Identification of Genetically Modified Maraba Virus as an Oncolytic Rhabdovirus

Brun, Jan, McManus, Dan, Lefebvre, Charles, Hu, Kang, Falls, Theresa, Atkins, Harold, Bell, John C, McCart, J. Andrea, Mahoney, Douglas, Stojdl, David F

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 493 KB

english, 2010

247

Optimization of Targeted Cell Replacement Therapy: A New Approach for Lung Disease

Duchesneau, Pascal, Wong, Amy P, Waddell, Thomas K

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 346 KB

english, 2010

248

Hyperactive Sleeping Beauty Transposase Enables Persistent Phenotypic Correction in Mice and a Canine Model for Hemophilia B

Hausl, Martin A, Zhang, Wenli, Müther, Nadine, Rauschhuber, Christina, Franck, Helen G, Merricks, Elizabeth P, Nichols, Timothy C, Kay, Mark A, Ehrhardt, Anja

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 529 KB

english, 2010

249

Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

Monahan, Paul E, Lothrop, Clinton D, Sun, Junjiang, Hirsch, Matthew L, Kafri, Tal, Kantor, Boris, Sarkar, Rita, Tillson, D Michael, Elia, Joseph R, Samulski, R Jude

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 257 KB

english, 2010

250

Gene Delivery of a Mutant TGFβ3 Reduces Markers of Scar Tissue Formation After Cutaneous Wounding

Waddington, Simon N, Crossley, Rachel, Sheard, Vicky, Howe, Steven J, Buckley, Suzanne MK, Coughlan, Lynda, Gilham, David E, Hawkins, Robert E, McKay, Tristan R

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 464 KB

english, 2010

251

Retraction to “Correction in Female PKU Mice by Repeated Administration of mPAH cDNA Using phiBT1 Integration System”

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 664 KB

english, 2010

252

Dystrophin Isoform Induction In Vivo by Antisense-mediated Alternative Splicing

Fletcher, Sue, Adams, Abbie M, Johnsen, Russell D, Greer, Kane, Moulton, Hong M, Wilton, Steve D

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 476 KB

english, 2010

253

Use of Adenoviral Vectors to Target Chemotherapy to Tumor Vascular Endothelial Cells Suppresses Growth of Breast Cancer and Melanoma

Li, Pingchuan, Liu, Yanzheng, Maynard, Jonathan, Tang, Yucheng, Deisseroth, Albert

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 500 KB

english, 2010

254

A High-throughput Pharmacoviral Approach Identifies Novel Oncolytic Virus Sensitizers

Diallo, Jean-Simon, Boeuf, Fabrice Le, Lai, Frances, Cox, Julie, Vaha-Koskela, Markus, Abdelbary, Hesham, MacTavish, Heather, Waite, Katherine, Falls, Theresa, Wang, Jenny, Brown, Ryan, Blanchard, Jan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 437 KB

english, 2010

255

Oncolytic Viruses: An Approved Product on the Horizon?

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 269 KB

english, 2010

256

Advances in the Field of Lentivector-based Transduction of T and B Lymphocytes for Gene Therapy

Frecha, Cecilia, Lévy, Camille, Cosset, François-Loïc, Verhoeyen, Els

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 255 KB

english, 2010

257

In Vivo RNAi-Mediated α-Synuclein Silencing Induces Nigrostriatal Degeneration

Gorbatyuk, Oleg S, Li, Shoudong, Nash, Kevin, Gorbatyuk, Marina, Lewin, Alfred S, Sullivan, Layla F, Mandel, Ronald J, Chen, Weijun, Meyers, Craig, Manfredsson, Fredric P, Muzyczka, Nicholas

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 614 KB

english, 2010

258

Expression of Human α1-Antitrypsin in Mice and Dogs Following AAV6 Vector-mediated Gene Transfer to the Lungs

Halbert, Christine L, Madtes, David K, Vaughan, Andrew E, Wang, Zejing, Storb, Rainer, Tapscott, Stephen J, Miller, A Dusty

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 7.69 MB

english, 2010

259

α-Synuclein Expression in Rat Substantia Nigra Suppresses Phospholipase D2 Toxicity and Nigral Neurodegeneration

Gorbatyuk, Oleg S, Li, Shoudong, Nha Nguyen, Frederic, Manfredsson, Fredric P, Kondrikova, Galina, Sullivan, Layla F, Meyers, Craig, Chen, Weijun, Mandel, Ronald J, Muzyczka, Nicholas

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 791 KB

english, 2010

260

Enhancement of Drug Delivery: Enzyme-replacement Therapy for Murine Morquio A Syndrome

Tomatsu, Shunji, Montaño, Adriana M, Dung, Vu Chi, Ohashi, Amiko, Oikawa, Hirotaka, Oguma, Toshihiro, Orii, Tadao, Barrera, Luis, Sly, William S

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.59 MB

english, 2010

261

RNA Silencing as a Natural Antiviral Defense System in Mammals: Where Are We Now?

Pfeffer, Sébastien

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

262

Effect of Genome Size on AAV Vector Packaging

Wu, Zhijian, Yang, Hongyan, Colosi, Peter

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 445 KB

english, 2010

263

Induction of Shock After Intravenous Injection of Adenovirus Vectors: A Critical Role for Platelet-activating Factor

Xu, Zhili, Smith, Jeffrey S., Tian, Jie, Byrnes, Andrew P.

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 233 KB

english, 2010

264

Targeting of p53-Transcriptional Dysfunction by Conditionally Replicating Adenovirus Is Not Limited by p53-Homologues

Kühnel, Florian, Gürlevik, Engin, Wirth, Thomas C, Strüver, Nina, Malek, Nisar P, Müller-Schilling, Martina, Manns, Michael P, Carnero, Amancio, Zender, Lars, Kubicka, Stefan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 552 KB

english, 2010

265

Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease

Mah, Cathryn S, Falk, Darin J, Germain, Sean A, Kelley, Jeffry S, Lewis, Melissa A, Cloutier, Denise A, DeRuisseau, Lara R, Conlon, Thomas J, Cresawn, Kerry O, Fraites Jr, Thomas J, Campbell-Thompson,

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 419 KB

english, 2010

266

In vivo Fluorescence Imaging of Muscle Cell Regeneration by Transplanted EGFP-labeled Myoblasts

Xu, Xiaoyin, Yang, Zhong, Liu, Qiang, Wang, Yaming

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 439 KB

english, 2010

267

AAV Vectors Avoid Inflammatory Signals Necessary to Render Transduced Hepatocyte Targets for Destructive T Cells

Somanathan, Suryanarayan, Breous, Ekaterina, Bell, Peter, Wilson, James M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 555 KB

english, 2010

268

AAV Provides an Alternative for Gene Therapy of the Peripheral Sensory Nervous System

Beutler, Andreas S

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 275 KB

english, 2010

269

Gene Correction by Homologous Recombination With Zinc Finger Nucleases in Primary Cells From a Mouse Model of a Generic Recessive Genetic Disease

Connelly, Jon P, Barker, Jenny C, Pruett-Miller, Shondra, Porteus, Matthew H

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 378 KB

english, 2010

270

Immunology and Gene Therapy: Shoulder to Shoulder Into the Fray

Smerdou, Cristian, Ochoa, Carmen, Quetglas, Jose I, Fontanellas, Antonio, Gonzalez-Aseguinolaza, Gloria, Vile, Richard G, Melero, Ignacio

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 244 KB

english, 2010

271

Defining the Probability that a Cell Therapy Will Produce a Malignancy

Prockop, Darwin J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 194 KB

english, 2010

272

POD Nanoparticles Expressing GDNF Provide Structural and Functional Rescue of Light-induced Retinal Degeneration in an Adult Mouse

Read, Sarah P, Cashman, Siobhan M, Kumar-Singh, Rajendra

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 410 KB

english, 2010

273

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 214 KB

english, 2010

274

Optimization of Peptide Nucleic Acid Antisense Oligonucleotides for Local and Systemic Dystrophin Splice Correction in the mdx Mouse

Yin, HaiFang, Betts, Corinne, Saleh, Amer F, Ivanova, Gabriela D, Lee, Hyunil, Seow, Yiqi, Kim, Dalsoo, Gait, Michael J, Wood, Matthew JA

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 940 KB

english, 2010

275

Rational Design of Micro-RNA-like Bifunctional siRNAs Targeting HIV and the HIV Coreceptor CCR5

Ehsani, Ali, Saetrom, Pål, Zhang, Jane, Alluin, Jessica, Li, Haitang, Snøve Jr, Ola, Aagaard, Lars, Rossi, John J

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 483 KB

english, 2010

276

114. Enhancement of the Immune Response of a Noval DNA Vaccine Encoding Conserved NS3 and Core Fusion Gene of HCV Injected by Intradermal Electrotransfer in Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

277

9. A Simplified Approach for Robust Minicircle DNA Vector Production Using a New Genetically-Modified E. Coli Producer Strain ZYCY10P3S2T

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 317 KB

english, 2010

278

135. Cell Autonomy in Methylmalonic Acidemia (MMA): Implications for Gene and Cell Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 406 KB

english, 2010

279

679. Rescue and Long-Term Outcome of a Murine Model of Methylmalonic Acidemia Following Liver-Directed, AAV-Mediated Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

280

2. Targeting Directed Evolution of AAV Capsids for Specific Therapeutic Applications

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 195 KB

english, 2010

281

3. Enhanced Hepatic Transgene Expression Following Intravenous Delivery of Novel Stem Cell-Derived Recombinant AAV Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 279 KB

english, 2010

282

5. Cell Entry Pathway Defines Efficiency and Specificity of rAAV Targeting Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 257 KB

english, 2010

283

7. Intravenously Administered rAAV9 Vector Efficiently Transduces Tissues In Vivo Via Caveolin-1-Independent Transport across the Capillary Endothelial Cell Barrier

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 257 KB

english, 2010

284

8. Directed Evolution of Adeno-Associated Virus Variants That Evade Human Neutralizing Antibodies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 375 KB

english, 2010

285

11. Zinc Finger Nucleases Drive Efficient Gene Disruption and Site-Specific Gene Addition in Human HSC

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

286

12. Manipulating piggyBac Transposon Chromosomal Integration Site-Selection in Human Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

287

13. Optimized Recovery of Integration Sites from Gene-Modified Cells Using Mu Transposition In Vitro

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 692 KB

english, 2010

288

14. An In Vitro-Identified High Affinity Nucleosome Positioning Signal Is Capable of Transiently Positioning a Nucleosome In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 680 KB

english, 2010

289

15. Hyperactive piggyBac -Mediated Gene Transfer in Somatic Cells In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 254 KB

english, 2010

290

16. Homing Endonucleases for Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 254 KB

english, 2010

291

17. AAV-Mediated Gene Therapy Restores Visual Function and Behavior to a Mouse Model of Leber Congential Amaurosis-1 (LCA1)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 282 KB

english, 2010

292

19. Brain Gene Therapy for Metachromatic Leucodystrophy: Towards Clinical Trial

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

293

20. Neonatal Intracerebroventricular Infusion of AAV2/8 Vector Leads to High Levels of Human α-L-Iduronidase Expression in the Brain and Complete Neurophenotypic Correction in a Murine Model of Mucopolysaccharidosis Type I

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

294

21. Circuit-Specific Expression of Channelrhodopsin Restores Visual Function in Blind rd1, rd16, and rho -/- Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

295

23. rAAV Delivery of α-Synuclein-Specific Single Chain Antibodies Attenuates Dopamine Loss in a Mouse Model of Parkinson's Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 219 KB

english, 2010

296

24. Patterns of Motor Function Recovery in a Murine Model of Severe Menkes Disease Rescued by Brain-Directed AAV5 Gene Therapy Plus Copper

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

297

25. Adoptive Transfer of Peripheral Blood Lymphocytes Genetically Modified To Express a T Cell Receptor Recognizing Carcinoembryonic Antigen into Patients with Metastatic Colorectal Cancer Induced Inflammatory Colitis without Mediating Anti-Tumor Effects

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 219 KB

english, 2010

298

26. Adenovirus Mediated Expression of Flt3L and HSV1-TK within the Brain Tumor Microenvironment Elicits Higher Therapeutic Efficacy Than Dendritic Cells’ Vaccination

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 219 KB

english, 2010

299

27. Pharmacokinetics of Anti-PSMA Gene-Modified T Cells Infused after Non-Myeloablative Conditioning in Prostate Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

300

28. Binary Control of Tumor-Specific Cytotoxic T Lymphocytes by Transgenic IL7 and IL7r Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

301

29. Multi-Tumor-Antigen-Specific Cytotoxic T Lymphocytes for Cancer Immunotherapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

302

30. Simultaneous Comparison of T Cells Expressing 1st or 2nd Generation CARs in Human Subjects with B-Cell Malignancies: Contribution of Costimulatory Endodomains

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

303

31. Prolonged Regression of Lymphoma and Elimination of CD19+ Cells in a Patient That Received Anti-CD19 Chimeric Antigen Receptor-Transduced T Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

304

32. Marked Regression of Adenopathy Following Infusion of Autologous T Cells Genetically Targeted to the CD19 Antigen in a Patient with Bulky CLL

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

305

33. Demonstration of Delivery and Antitumoral Activity of JX-594, a Targeted Multi-Mechanistic Oncolytic Poxvirus, Following a Single Intravenous Infusion in Patients with Refractory Metastatic Cancers

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

306

34. Highly Attenuated Vaccinia Virus with microRNA-Regulated Oncolysis for Cancer Virotherapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

307

35. A Phase 1 Trial of Intravesical (IVE) CG0070 in Patients with Superficial Bladder Cancer after BCG Failure

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

308

36. A Phase 1 Trial of the Novel Infectivity-Enhanced Bicistronic Adenovirus Ad5.SSTR/TK.RGD in Recurrent Gynecologic Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 263 KB

english, 2010

309

37. Insect Cell Carriers for Systemic Delivery of Oncolytic Viruses

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

310

38. Clinical Translational Development of rVSV(MD51)-M3: Biodistribution and Pharm/Tox Studies in Rats and Monkeys

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

311

39. A High-Throughput Pharmaco-Viral Approach Identifies Oncolytic Novel Virus Sensitizers

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

312

40. Identification of Adenovirus Modulator Genes in Human Cancer Cells by Whole Genome RNAi Library Screening

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

313

41. Repeated Autologous Infusions of Cells Modified with VSV-G-Pseudotyped Lentiviral Vector VRX496 Can Induce Anti-VSV-G Humoral Response in Humans without Clinical Consequence or Effect on Modified Cells Persistence: Findings from Lexgenleucel-T Phase 2 Clinical Trial

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 264 KB

english, 2010

314

43. Induction of Regulatory T Cells as an Approach To Modulate CTL Responses Against AAV Capsid Epitopes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

315

44. Long-Term Phenotypic Correction of Hemophilia A after Neonatal Administration of AAV

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

316

46. Autologous Dendritic Cell Therapy for Tolerance Induction

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

317

47. Differential Transgene Expression in Dendritic Cells with Unique Regulatory Cassettes Impacts Efficacy in Pompe Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

318

48. Impact of the Host Environment on the Control of Gene Transfer-Induced Immune Responses with miRNA142.3p-Regulated Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

319

49. Systemic Delivery of Antisense Morpholino Corrects RNA Mis-Splicing and Reduces Myotonia in a Transgenic Mouse Model of Myotonic Dystrophy Type 1

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

320

51. Using rAAV Delivered miRNAs To Knockdown Misfolded Human Alpha 1 Antitrypsin in a Transgenic Mouse Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

321

52. Constitutive Long-Term Expression of shRNA in Liver Induces Cellular Stress

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 235 KB

english, 2010

322

53. In Vivo AAV Delivered Allele Specific shRNA for the Knockdown of Alpha-1 Antitrypsin

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

323

54. Expression Determinants of Mammalian Argonaute Proteins in Mediating Gene Silencing

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

324

55. Antisense Oligonucleotide-Mediated Therapy Increases SMN Levels in the CNS and Prolongs Survival in a Severe Mouse Model of Spinal Muscular Atrophy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

325

56. Multiple Exon-Skipping Using Cell-Penetrating Morpholinos for Dystrophic Dogs

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

326

57. Enveloped AAV Vectors for Gene Transfer Applications

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

327

59. Gene Delivery to the Pig Cone-Enriched Retina Highlights Species-Specific Differences in AAV-Mediated Photoreceptor Transduction

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

328

61. Tyrosine Mutations in the Capsid of AAV Vectors Alters Transduction Properties in the Retina

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

329

62. Riding Along with AAV: Mechanistic Studies on Vesicular Trafficking Pathways of AAV Serotypes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

330

64. Efficient Transduction of Mouse Liver and Other Organs Following Intravenous Administration of a Recombinant Porcine AAV

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

331

65. Rational Design Meets Directed Evolution for Tailoring the AAV Capsid

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

332

66. AAV5 Transduction and Transgene Expression and Bioactivity in Fibroblast-Like Synoviocytes from Rheumatoid Arthritis Patients

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

333

67. Pre-Injection of Polymers Can Increase the In Vivo Transduction Efficiency of Adeno-Associated Virus Type 2

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

334

68. Therapeutic Expression of FIX at Reduced Doses Using AAV-Protein Phosphotase-5 Helper Virus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 308 KB

english, 2010

335

69. A Scalable Process for Purification of Recombinant Adeno-Associated Virus Serotype 9

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 281 KB

english, 2010

336

70. Adeno- Associated Virus Type 2 Rep Proteins Mediate Preferential Integration of a Lentiviral Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 281 KB

english, 2010

337

71. High Throughput Assay for Quantifying In Vitro AAV Vector Transduction Efficiency in ARPE19 Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 300 KB

english, 2010

338

72. Optimization of Parameters for AAV Production in Insect Cells and Conditions for Baculovirus Storage

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 281 KB

english, 2010

339

73. Radiation-Induced Liver Fibrosis Is Mitigated by Gene Therapy Against Transforming Growth Factor-ß Signaling in the Rat

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 281 KB

english, 2010

340

74. Protection of Salivary Glands from Irradiation Damage by hMnSOD Gene Transfer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 306 KB

english, 2010

341

76. Virion-Associated Co-Factor HMGB1 Facilitates Transposition from the HSV/Sleeping Beauty Amplicon Vector Platform

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

342

77. hKGF Gene Transfer for the Prevention of Radiation-Induced Salivary Hypofunction

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 246 KB

english, 2010

343

78. Generation of Replication-Defective Adenovirus Vector Expressing HCV Structural Gene CE1E2 and Comparative Immunogenicity in Mice with Different Delivery Routes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

344

79. Interaction between Adenoviral Vector-Transduced Dendritic Cell-Based Cancer Vaccine and T Regulatory or T Helper 17 Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

345

80. Replicating Adenoviruses That Harbors the ADP Protein and the Sodium Iodide Symporter Protein (NIS) under the Control of the Major Late Adenovirus Promoter

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 205 KB

english, 2010

346

82. Sustained Inhibition of Hepatitis B Virus Replication In Vivo Using Helper-Dependent Adenovirus Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 205 KB

english, 2010

347

83. Adenovirus Serotype 5/35 Is Excellent for Genetic Engineering of Tumor-Directed T Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 223 KB

english, 2010

348

84. Bipartite Adenoviral Vector Encoding hHGF and hXIAP for Improved Human Islet Transplantation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 205 KB

english, 2010

349

85. Vector Prime-Protein Boost Vaccine for Patients with Therapy Induced Lymphopenia

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 205 KB

english, 2010

350

86. The Effect of Surface Modification of Adenovirus with an Arginine-Grafted Bioreducible Polymer on Transduction Efficiency and Immunogenicity in Cancer Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

351

87. Safe and Efficient Delivery of Meganucleases for Lentivirus-Mediated Gene Targeting

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

352

88. Site-Specific Genome Modification of Human Primary T Lymphocytes To Improve the Safety and Efficacy of Adoptive Immunotherapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 245 KB

english, 2010

353

89. Single-Strand Nicks Induce Homologous Recombination with Less Toxicity Than Double-Strand Breaks Using an AAV Template

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

354

90. Targeted Gene Insertion in Human Chromosomes Using a Homing Endonuclease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

355

94. Targeted Plasmid Integration into the PiggyBac Transposon by an Engineered Zinc Finger Recombinase

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

356

95. Development of Autonomously Working Zinc-Finger Nuclease Pairs for Targeted Genome Engineering

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

357

96. Adeno-Associated Virus-2 (AVV2) Integrase Mediates Specific Integration to Locus AAVS1 on Chromosome 19q13.3 in a Hematopoietic Cell Line

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 293 KB

english, 2010

358

97. Gene Correction in Mouse Liver

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

359

98. A Transient Cold Shock Enhances Zinc-Finger Nuclease-Mediated Gene Disruption

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

360

99. Single-Polarity rAAV May Enhance Hepatic Gene Targeting In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

361

100. Over-Expression of Adiponectin and Adiponectin Receptor 2 Gene by the Method of Hydrodynamic Gene Delivery Prevents Weight Gain and Development of Insulin Resistance in AKR/J Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 275 KB

english, 2010

362

101. Duration of Expression after Gene Electrotransfer to Skin Depends on the Gene of Interest

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

363

102. Intravenous Delivery of pDNA and siRNA into Muscle by Ultrasound-Mediated Bubble Liposomes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

364

103. Prototype Development and Pre-Clinical Immunogenicity Analysis of a Novel Minimally Invasive Electroporation Device

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 415 KB

english, 2010

365

104. Ultrasound-Assisted Non-Viral Gene Transfer to the Salivary Glands

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 414 KB

english, 2010

366

105. Development of Electroporation Formulation for Cell Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 414 KB

english, 2010

367

106. Ca2+ Electrotransfer; a Quick Stop Procedure Against Transgenic Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 243 KB

english, 2010

368

107. In Vivo Gene Delivery to the Skin by Electroporation Using a Multiple Electrode Array

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 243 KB

english, 2010

369

109. Efficiency of Electroporation Delivery of Peptide Nucleic Acid (PNA) Depends on PNA Charge and Electrotransfer Protocol

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 273 KB

english, 2010

370

110. Biodistribution of Plasmid AMEP, a Potent Antitumor Gene Product for the Treatment of Metastatic Melanomas

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

371

111. In Vivo Intracellular Injection Needle (IvIn) Technology: A New Needle for Improved Cellular Uptake of DNA in a Live Animal

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

372

113. Toxicology and Safety Studies of Plasmid AMEP, a New Efficient Antitumor Gene Product for the Treatment of Metastatic Melanomas

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

373

115. Electroporation-Enhanced DNA Prime/Protein Boost Strategy To Improve Cross-Clade Immunogenicity of HIV-1 Envelope Antigen

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 287 KB

english, 2010

374

116. Effectiveness of Impedance Spectroscopy To Monitor Impact of an Electroporation Protocol on Tissue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 287 KB

english, 2010

375

118. Biodegradable Polyamino Acid-Based Polycations for Safe and Effective Gene Transfer Minimizing Cumulative Toxicity

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 306 KB

english, 2010

376

119. Interplay of Clathrin and Caveolin Trafficking Pathways in Polymer-Based Gene Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

377

120. Folate Linked Targeting Systems Increase Transfection Efficiency and Specificity in Human Epithelial Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 263 KB

english, 2010

378

121. In Vivo Gene Delivery from Modified PLG Scaffolds

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

379

122. The Influence of Charge Density and Substituent Composition on Imidazolium-Containing Polymers for Mediated DNA Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

380

123. Concentrated PEI Formulations Retain Biological Efficacy Following Long-Term Storage at 4°C

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 361 KB

english, 2010

381

124. Charge Density and Molecular Weight of Polymeric Gene Carrier Are Key Parameters Influencing Its DNA Compaction Ability and Transfection Efficiency

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 342 KB

english, 2010

382

125. Designing Imidazole-Containing Polymeric Vectors with Controlled Charge Densities for Gene Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 342 KB

english, 2010

383

126. Bioreducible Poly(amido ethyleneimine) and Its Polyethylene Glycol Graft Copolymer Analog Formulations Offer Ease of Use in Optimizing Improved Nucleic Acid Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

384

127. Role of Glycosaminoglycans in Peptide-Mediated DNA Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

385

128. Galactosylated-DPC Nanoparticles for Targeted Hepatic Gene Delivery In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 365 KB

english, 2010

386

129. Effect of Molecular Weight on Plasmid DNA Delivery Efficiency of Palmitic Acid Substituted Polyethylenimine

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 346 KB

english, 2010

387

131. A Novel Cationic Lipophosphoramide with Phytanyl Chains: Gain in Transfection Efficiency and Clinical Approach Concerning the Response to Lipofection

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

388

132. Cationic Peptide-Based DNA Delivery In Vitro: The Role of DNA Condensation and Release

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

389

133. Modeling and Correcting Fibrous Dysplasia with Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 397 KB

english, 2010

390

134. Bone Marrow Transplantation Cures Hemophilia A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 370 KB

english, 2010

391

137. Development of a Safe Hematopoietic Stem Cell (HSC) Mobilization Strategy for Gene Therapy of Thalassemia

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 408 KB

english, 2010

392

138. Long Term Expression in Liver from scAAV Seems Derived from Integrated Vector Genomes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 389 KB

english, 2010

393

139. Human Amnion Epithelial (AE) Stem Cell Transplant Improves Disease Phenotype and Survival in Mouse Models of Intermediate Maple Syrup Urine Disease (MSUD)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 389 KB

english, 2010

394

140. Gene Therapy Approaches Combining the Immunomodulatory Properties of Liver and IGF-I To Prevent Type I Diabetes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

395

141. Hepatocyte Engineering and Genetic Modification Approaches of Hemophilia Hepatocytes within a Living Mouse

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

396

142. Serotype Optimization of an AAV Vector for Gene Therapy in Canine and Murine Glycogen Storage Disease Type Ia (GSD-Ia)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

397

143. Systemic Delivery of Bioactive Glucagon-Like Peptide 1 Following Adenoviral Vector-Mediated Gene Transfer to Murine Submandibular Glands

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

398

144. A Non-Viral, GNE-Lipoplex Treatment to Correct Sialylation Defects in Gne -Mutant (M712T) Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 264 KB

english, 2010

399

145. Lentivirally Induced Over-Expression in the Mouse Hypothalamus; a Study of the Effects of Melanin Stimulating Hormones (MSH) in Obesity and Diabetes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 197 KB

english, 2010

400

146. Alternative Methods for Ex Vivo Gene Transfer to Primary Murine B Cell

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 197 KB

english, 2010

401

147. A Full Characterization of the CAG 140 Huntington's Disease Mouse Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 216 KB

english, 2010

402

148. Assessment of Pre-Clinical Safety of GMP Grade GNE-Lipoplexes after Repeat Intravenous Injections in Balb/c Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 197 KB

english, 2010

403

149. Pre-Existing Antibodies to AAV8 Attenuates Micro-Dystrophin Expression Following Targeted Vascular Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 197 KB

english, 2010

404

150. AAV Microgene Transfer Reveals the Cellular Motif for Dystrophin-Mediated Sarcolemmal Neuronal Nitric Oxide Synthase (nNOS) Localization

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

405

151. Cell Therapeutic Approach to Duchenne Muscular Dystrophy Using Myogenic Differentiation of Multipotent Mesenchymal Stromal Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

406

152. Splice Site Strength and Nonsense-Associated Exon Skipping in the DMD Gene

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

407

154. Exon Exchange Approach To Repair Duchenne Dystrophin Transcripts

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

408

155. Gentamicin Treatment of Duchenne Muscular Dystrophy Reinforces the Potential for Mutation Suppression Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 264 KB

english, 2010

409

156. Reverse Perfusion Affords Efficient Whole-Body Gene Transfer to Muscle in the Large Animal

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

410

158. Genetic Correction of Netherton Syndrome Keratinocyte Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

411

159. Adenoviral Human Vascular Endothelial Growth Factor-D Gene Transfer Causes Angiogenesis and Growth and Enlargement of Lymphatic Vessels in Ischemic Mouse Hind Limbs

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

412

160. Validation of Intravascular Administration of Adeno-Associated Vvirus for Muscular Dystrophies in a Non-Human Primate Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 254 KB

english, 2010

413

161. Suppression of Cocaine-Induced Hyperactivity in Mice Pre-Treated with an Adenovirus-Based Anti-Cocaine Vaccine

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

414

162. Translation of Electroporation-Mediated DNA Vaccine Delivery into a Phase I Clinical Trial Evaluating an HIV DNA Vaccine in Healthy Volunteers

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

415

163. Longterm Protective Immunity Against Lethal Pulmonary Challenge with the Pseudomonas aeruginosa Following Mucosal Administration of AdC7-OprF Expressing the P. aeruginosa Protein OprF

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

416

164. Characterization of Adenoviruses Shed from the Macaque Gut and Evaluation of Immune Responses to Adenoviruses in the Macaque in the Context of Future Adenoviral Vaccine Studies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

417

165. A Lentiviral HIV Vaccine Candidate Elicits Long-Term Humoral and Cellular HIV-Specific Immunity and Minimal Neutralizing Activity

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 334 KB

english, 2010

418

166. Coexpressed RIG-I Agonist Enhances Humoral Immune Response to Influenza DNA Vaccine

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 334 KB

english, 2010

419

167. Genetically Engineered Bifidobacterium Displaying Salmonella -Antigen Protects Mice from Lethal Challenge of Salmonella Typhimurium in a Murine Typhoid Fever Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 353 KB

english, 2010

420

168. Signaling Lymphocytic Activation Molecule (SLAM, CD150)-Blind Measles Virus Is Attenuated and Induces Strong Adaptive Immune Responses in Rhesus Monkeys

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 278 KB

english, 2010

421

169. Evaluation of Mismatched Immunity To Expand Cross-Protection Against Influenza Virus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 278 KB

english, 2010

422

170. Immune Response Against Measles Virus Encoding Helicobacter pylori Neutrophil-Activating Protein Antigen

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 306 KB

english, 2010

423

171. Generation of Chimeric GB Virus B Clones Encoding the Hepatitis C Virus E1E2p7 and coreE1E2p7 toward the Infected Marmoset Models

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

424

172. Characterization of Immune Response in Mice Induced by Recombinant Vaccinia Virus (Tiantan) Based Multivalent H5N1 Avian Influenza Vaccines

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

425

173. The Effects of the Calreticulin on the Immune Response Induced by DNA Vaccine Containing the Classical Swine Fever Virus E2 Gene in Mouse Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 280 KB

english, 2010

426

174. Evaluation of the Multi-Electrode Array for Use in DNA Vaccination Against Bacillus anthracis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

427

175. Enhancing the Priming of Tumor Antigen Specific T Cell Responses Using Oncolytic Viruses

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

428

176. AdCD40L Immunogene Therapy for Bladder Carcinoma – A Phase I/IIa Trial

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

429

177. Treatment of Refractory Cancer Patients with a Serotype 5/3 Chimeric Oncolytic Adenovirus Expressing Granulocyte Macrophage Colony-Stimulating Factor (GM-CSF): Virological, Immunological and Clinical Endpoints

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

430

179. PiggyBac Transposon Modified EBV Specific Human T Lymphocytes for HER2-Positive Cellular Immunotherapy In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 249 KB

english, 2010

431

180. Adenovirus-Mediated Intratumoral Expression of Immunostimulatory Molecules Together with Systemic Depletion of Regulatory T-Cells Induces Tumor-Destructive Immune Responses in Mouse Models

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

432

182. Genetically Engineered Malignant Glioma-Specific T Cells Resistant to Glucocorticoids

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 245 KB

english, 2010

433

184. Adoptive Transfer of Autologous T Cells Targeted to Prostate Specific Membrane Antigen (PSMA) for the Treatment of Castrate Metastatic Prostate Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 265 KB

english, 2010

434

185. A New Human-Derived Reporter Gene Suitable for Clinical PET Imaging of T-Cell Trafficking

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 245 KB

english, 2010

435

187. Development of High-Performance HVJ-E for Cancer Immune Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 294 KB

english, 2010

436

190. Development of an HLA-A*0201 Restricted T Cell Receptor That Recognizes Peptide Epitopes from MAGE-A3 and MAGE-A12 for Targeted Adoptive T Cell Immunotherapy of Multiple Cancer Types

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

437

191. A Comparative Study of Stem Cell-Based Cell Carrier Systems for the Systemic Delivery of an Oncolytic Adenovirus for Antiglioma Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

438

192. A Novel Coxsackievirus B Shows Remarkable Oncolytic Capacity Against Lung Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

439

193. Preclinical and Clinical Data with a Fully Serotype 3 Oncolytic Adenovirus Ad3-hTERT-E1A in the Treatment of Advanced Solid Tumors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 196 KB

english, 2010

440

194. Mechanism of Tumour Penetration by Oncolytic Viruses – Investigation in a 3 Dimensional In Vitro Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 196 KB

english, 2010

441

197. Safety and Immunological Effects of Corticosteroids in Patients Treated with Oncolytic Adenoviruses

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 196 KB

english, 2010

442

198. Effect of Decorin on Overcoming the Extracellular Matrix Barrier for Oncolytic Virotherapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

443

199. Chondroitinase ABC1 Mediated Enhancement of OV Spread and Efficacy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

444

200. Use of PET Imaging for In-Vivo Monitoring of Replication Competent Retrovirus (RCR) Transduction of Malignant Gliomas

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

445

201. Systemic Cancer Screening Using an Oncolytic HSV To Induce Tumor-Selective Expression of a Secretable Serum Biomarker

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

446

202. Therapeutic and Tumor-Specific Immunity Induced by Combination of Dendritic Cells and Oncolytic Adenovirus Expressing IL-12 and 4–1BBL

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

447

203. Oncolytic Herpes Simplex Virus Encoding Interferon ß Gene for Squamous Cell Carcinoma of the Head and Neck

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 376 KB

english, 2010

448

204. Targeted Suicide Gene Therapy with Oncolytic Adenovirus Coding for FCU1, a Fusion Protein of Cytosine Deaminase and Uracilphosphoribosyltransferase

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 328 KB

english, 2010

449

205. Controlled Nuclear Localization of p53 in Breast Cancer Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 328 KB

english, 2010

450

206. Effect of Transcriptionally Targeted Suicide Gene Therapy for Small Cell Lung Cancer In Vitro and In Vivo by DOTAP/Cholesterol- and Adenoviral-Mediated Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

451

207. NF-κB Activity Regulates MSC Accumulation at Tumor Sites

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

452

208. Dynamics of Therapy of Malignant Melanoma with Vesicular Stomatitis Virus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 308 KB

english, 2010

453

209. Therapeutic Efficacy of a Lentiviral Mediated Thymidine Kinase/Ganciclovir Treatment in an Immunocompetent Rat Malignant Glioma Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 282 KB

english, 2010

454

210. Insertion of Transcription Factor Nuclear Factor Kappa B Binding Sites into Plasmid DNA Expressing a Suicide Gene Facilitates Increased Small Cell Lung Cancer Cell Death In Vitro Due to Increased Nuclear Uptake

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 282 KB

english, 2010

455

211. Preirradition Gene Therapy Against Transforming Growth Factor-ß Signal Is Radioprotective for the Liver

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 302 KB

english, 2010

456

212. Rabbit VX-2 Brain Tumor Model for Development of Improved Vector Administration Techniques

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 282 KB

english, 2010

457

213. Nano-Flow 2D-HPLC MALDI-TOF MS/MS for Differentially Expressed Protein Analysis in Breast Cancer Patient Samples

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 282 KB

english, 2010

458

214. In Vitro Characterization for In Vivo Translation of a Novel Non-Viral Gene Delivery Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 479 KB

english, 2010

459

215. Altering the Tropism of Herpesvirus saimiri to Selectively Infect Hepatocellular Carcinoma (HCC) Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 399 KB

english, 2010

460

216. Improved Gene Transfer into Renal Carcinoma Cells Using an Adenovirus Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 399 KB

english, 2010

461

217. Prostate Cancer Gene Therapy Using Adenovirus Expressing a Novel Apoptosis-Inducing Gene and Its Conjugation with Chemotherapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

462

218. Development and Preclinical Testing of Large Batches of SNS01-A – An eIF5A-Based Gene Therapy Nanoparticle Designed for the Treatment of Multiple Myeloma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

463

219. Angiotensin II Type 2 Receptor Gene Mediated Apoptosis of Human Lung Adenocarcinoma A549 Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 281 KB

english, 2010

464

221. Use of the Natural Human Artemis Promoter To Regulate Human Artemis Expression Prevents Toxicity and Corrects Artemis Deficient SCID after Ex Vivo Lentiviral Transduction in a Murine Model of the Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

465

222. Lack of Enhancer-Blocking Activity by 400bp cHS4 Insulator in a Human T- Lymphoid Cell line

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

466

223. Liver-Directed Intravascular Fetal Delivery of scAAV Facilitates Sustained Transgene Expression with Evidence of Transplacental Trafficking and Possible Vector Integration Events

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

467

224. Helper-Dependent Adenoviral Vector Gene Therapy Mediates Long-Term Low-Level Correction of Hemophilia A in the Murine Model after Neonatal Gene Transfer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 269 KB

english, 2010

468

225. Lentiviral Vector Mediated Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome: In Vitro and In Vivo Preclinical Studies with Human CD34+ Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 269 KB

english, 2010

469

227. Development of a Gene Transfer System for Wiskott-Aldrich Syndrome Using Foamy Virus Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 268 KB

english, 2010

470

228. Integration Site Distribution in Mice Following Therapeutic Gene Transfer To Treat Beta-Thalassemia

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 296 KB

english, 2010

471

229. Validation of the Tranduction of Patients Hematopoietic Progenitor Cells with a Clinical-Grade Lentiviral Vector Developed for Wiskott-Aldrich Syndrome Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

472

230. Generation of an Optimized Lentiviral Vector Encoding a High-Expression fVIII Transgene for Gene Therapy of Hemophilia A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

473

231. Biodistribution of a High Dose of Intraarticularly Administered AAV-5 Vectors to Lewis Rats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

474

233. Signed Outside: A Surface Marker System for Transgenic Cytoplasmic Proteins

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

475

234. Low Levels of Expression of CD18 Using the pgk Promoter in a Foamy Viral Vector To Treat Animals with Canine Leukocyte Adhesion Deficiency

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 295 KB

english, 2010

476

236. A Novel Human Artificial Chromosome Vector Derived from Chromosome 14 (14HAC): Higher Transgene Expression System Using Sub-Telomere Sequences

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 275 KB

english, 2010

477

237. Effective Gene Delivery to Wounds by Transplantation of Minced Skin Particles Transfected with Adenoviral-VEGF

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

478

238. MicroRNA-122 Regulation Prevents Liver Toxicity of Suicide Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

479

239. Regulation of Endogenous GAD67 Expression Using Artificial Transcription Factors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

480

240. Enhanced In Vivo Transgene Expression by Two-Step Transcription Amplification

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 284 KB

english, 2010

481

241. Targeted Knockdown of Serum Response Factor in Hearts of Adult SRF Conditional KnockOut Mice by AAV9-Mediated Transfer of Cre-Recombinase

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

482

242. Pro-Proliferative Functions of Drosophila Small Mitochondrial Heat Shock Protein 22 in Human Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

483

243. Interleukin-(IL)-24 Regulates Epithelial to Mesenchymal Transition (EMT) Transcription Factors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

484

244. Transient Epigenetic Gene Therapy as a Rising Arm of a Universal Stem Cell Gene Therapy Platform both for Ex Vivo and In Vivo Applications

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

485

245. The Application of “Bi-Functional” Short Hairpin RNA in A Furin-shRNA and GM-CSF (FANG) Autologous Vaccine for Cancer Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 266 KB

english, 2010

486

246. A pH-Sensitive Fusogenic Peptide Modified siRNA-Encapsulating Nanoparticles Exerts the Gene Silencing Synergistically with Tumor-Specific Cleavable PEG-Lipid

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

487

248. Augmentation of Docetaxel Anti-tumor Activity by a Stathmin-Specific Bi-Functional shRNA

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

488

249. RNAi- and U1i-Mediated Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

489

250. Lentiviral Delivery of EzH2 shRNA Inhibits Tumor Growth of Epithelial Ovarian Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

490

251. Prostate Tumor Radiosensitization through PSMA-Aptamer-Targeted siRNA Knock-Down of DNA Repair Pathways

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

491

252. Preclinical Safety and Toxicity Assessment of Stathmin 1 Bifunctional shRNA Formulation Following a Single Intravenous Injection in Rats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

492

253. Application of siRNA-Containing Nanoparticles for In Vitro and In Vivo Gene Silencing in Tumor by Controlling Intracellular Trafficking

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

493

254. The Novel Comb Shaped Amphiphilic Polyallylamine for Potential Oral Delivery of siRNA to Gastrointestinal Cancers: Physicochemical Characterizations and In Vitro Evaluations

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

494

255. Optimalization of Polymerase II sh/miRNA Expression for Therapeutic Applications

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

495

256. The Effect of Helper Lipids on Cationic Lipid-Based siRNA Delivery System

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 292 KB

english, 2010

496

257. c-Cbl shRNA-Expressing Adenovirus Functions as a Sensitizer of TRAIL-Induced Apoptosis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 253 KB

english, 2010

497

258. ncRNAs Originating from the Dystrophin Gene as Biomarker for Assessing Antisense Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 253 KB

english, 2010

498

259. Efficient In Vivo Delivery of Cholesterol-Conjugated siRNA to the Liver Using Endogenous Chylomicron Remnant

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 451 KB

english, 2010

499

260. Controlled Nonviral Gene Delivery and Expression Using Stable Neural Stem Cell Line Transfected with a Hypoxia-Inducible Gene Expression System

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 433 KB

english, 2010

500

261. Non-Viral Carriers Deliver mRNA Encoding Marker Proteins in HeLa cells and mRNA Encoding CXCR4 in Mesenchymal Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 462 KB

english, 2010

501

262. Genetic Modification of Neural Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

502

263. APOA-1 Is a Novel Marker of Erythroid Cell Maturation from Hematopoietic Stem Cells in Mice and Humans

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

503

264. A Stem Cell Gene Therapy Approach for Enhancing the Efficacy of Breast Cancer Therapy by Trastuzumab (Herceptin)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

504

266. SB-T-1214 Dramatically Downregulates Stem Cell-Related Gene Expression Profiles in Metastatic Colon Cancer Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

505

267. Identifying Genes and Signaling Pathways Regulating the Quiescent State in Muscle Stem (Satellite) Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 489 KB

english, 2010

506

268. Pathway Analysis of Multipotent Mesenchymal Stromal Cells Using BacMam Tools

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 452 KB

english, 2010

507

269. The Placental Microvasculature Is a Rich Source of Highly Proliferative Endothelial Colony Forming Cells Capable of Developing Functioning Vessels In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 453 KB

english, 2010

508

270. microRNA Function during Human Mesenchymal Stem Cell Adipogenic Differentiation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 644 KB

english, 2010

509

272. The Stromal Vascular Fraction of Human Term Placenta Is a Rich Source of Pluripotent Mesenchymal Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 467 KB

english, 2010

510

273. The HSV-2 Protein, ICP10PK, Potentiates the Neuroprotective Activity of Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

511

275. TAG Vaccine Clinical Trial Manufacturing

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 270 KB

english, 2010

512

276. Development and GMP Validation of Large Scale Lentiviral Transduction of CD34+ Cells for Metacromatic Leukodystrophy and Wiskott-Aldrich Syndrome Gene Therapy Clinical Trials

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 250 KB

english, 2010

513

277. Large Scale rVSV Viral Vector Production in a cGMP Facility

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 287 KB

english, 2010

514

278. Challenges in the Manufacturing of Retroviral Gene Therapy Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

515

279. Production of cGMP Grade Lentiviral Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

516

280. Design and Operation of a New State-of-the-Art Multi-Product cGMP Cleanroom Facility for Vector Production and Cell Manipulation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 257 KB

english, 2010

517

281. Highly Efficient Manufacturing of Clinical Grade Lentiviral Vector in a New, State of the Art Academic Good Manufacturing Practice (GMP) Facility

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

518

282. The Role of JNK in Lentivirus Gene Transfer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

519

284. A Novel Lentiviral Expression System Reveals Patchwork Colony Formation during Reprogramming to Induced Pluripotent Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

520

285. Targeted Genomic Modification and Inactivation of the CCR5 Gene Using PNA-Induced Homologous Recombination

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

521

286. Determining the Specificity of Zinc Finger Nucleases by Analyzing Insertion Site Distribution of Integrase Defective Lentiviral Vectors (IDLV) Trapped into DNA Double Strand Breaks

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

522

287. Systemic Administration of Dendrimer- and Aptamer-siRNA Conjugates Efficiently Suppresses HIV-1 Infection

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

523

288. CASPALLO: Phase I Clinical Trial of Allodepleted T Cells Transduced with Inducible Caspase 9 Suicide Gene after Haploidentical Stem Cell Transplantation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

524

290. Sustained Alpha-Sarcoglycan Gene Expression in LGMD2D Following Gene Transfer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

525

291. Advanced Generation RGD-Modified Oncolytic Adenovirus ICOVIR-7 in the Treatment of Cancer Refractory to Other Modalities

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

526

292. Final Results of Phase I Trial of GMCSF Gene-TGFß2 Antisense Gene Autologous Tumor Cell (TAG) Vaccine in Advanced Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 359 KB

english, 2010

527

293. Administration of Tumor-Specific Cytotoxic T Lymphocytes Engineered to Resist TGF-ß to Subjects with EBV-Associated Lymphomas

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 301 KB

english, 2010

528

294. Efficient Engraftment of MGMTP140K Gene-Modified CD34+ Cells Following Nonmyeloablative BCNU Conditioning in Patients with Glioblastoma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 320 KB

english, 2010

529

295. Thymic Renewal and Anti-Leukemic Effect in Adults after Haplodentical Transplantation and Suicide Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

530

296. Update on a Clinical Gene Therapy Trial for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

531

297. Sensitive In Vivo Models To Assess the Risk of Insertional Mutagenesis in the Liver upon Vector Systemic Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 264 KB

english, 2010

532

298. Systemic Injection in a Tumor Prone Mouse Unravels the Residual Genotoxicity of Self-Inactivating Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

533

299. Specific Insertions of Zinc Finger Domains into Gag-Pol Yields Engineered Retroviral Vectors with Selective Integration Properties

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

534

300. The D4Z4 Insulator Protects Retrovirus Vectors from Silencing in Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

535

301. Expression of the LEDGF Integrase Binding Domain (IBD) Severely Restricts HIV-1 Replication and Drives Evolution of Phenotypically Distinctive Integrase Mutants

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

536

302. Transient and Highly Efficient Delivery of Sleeping Beauty Transposase and Flp Recombinase mRNA in Target Cells Via RT-Deficient Retroviral Particles

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

537

303. Erasable Non-Integrating RNA Replicon Vectors for Generation of Immaculate iPS Cells Free from Transgenes and Sequence-Interference

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

538

304. Self-Inactivating Alpharetroviral Vectors with a Split-Packaging Design

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

539

305. Dissecting rAAV Integration Frequency in Non-Human Primates by Deep Sequencing

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

540

306. Non-Canonical Integrating AAV Vectors as a Safe Tool for the Future of Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 234 KB

english, 2010

541

307. Interaction with Homologous Recombination Pathways Leads To Loss of Functional Recombinant AAV (rAAV) Genomes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

542

309. A Self-Complimentary rAAV Vector Genome but Not Variations in the Capsid Sequence Magnifies the Innate Immune Response Following Liver Directed Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

543

310. A Proteomic Analysis of the Protein Composition in Recombinant AAV Vector Preparations

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

544

311. Downregulation of DNA-Damage Response Proteins in Postmitotic Tissues Correlates with Increased AAV Transduction

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 244 KB

english, 2010

545

312. Innate Immune Response towards Adeno-Associated Viral (AAV) Vectors in Human Liver Non-Parenchymal Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

546

313. Towards a Safe and Effective Gene Therapy Approach for Globoid Cell Leukodystrophy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

547

314. Mesenchymal Stem Cell Therapy for the Twitcher Mouse Model of Krabbe's Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

548

316. Therapeutic Potential of Genetically Modified Neural Stem Cells (NSCs) in a Mouse Model of Globoid Cells Leukodystrophy (GLD)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 244 KB

english, 2010

549

317. Adeno-Associated Virus and Tetanus Toxin Fragment C Based Vectors for Gene Therapy in the CNS

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 216 KB

english, 2010

550

318. Lineage Specific Lentiviral Mpl Expression Rescues Hematopoietic Stem Cell Defects in Mpl-/- Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 216 KB

english, 2010

551

319. Safety of Gene Therapy for Thalassemia

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 216 KB

english, 2010

552

320. Long-Term Phenotypic Correction of Skin by Fetal Gene Transfer in a Lethal Murine Model of Epidermolysis Bullosa

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 216 KB

english, 2010

553

321. Bile Duct Infusion of Plasmid DNA Mediated High Level of Transgene Expression in Rat Liver

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 578 KB

english, 2010

554

322. Image-Guided, Computer-Controlled Hydrodynamic Gene Delivery to Liver Results in Therapeutic Level of Human Alpha-1 Antitrypsin in Pigs and Dogs

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 544 KB

english, 2010

555

323. Targeting Magnetic Nanoparticles Impregnated with Adenovirus to Stented Arteries Using Uniform-Field Induced Magnetization in the Rat Carotid Model of Restenosis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 562 KB

english, 2010

556

324. Ultrasound-Activated Microbubbles for Gene Transfer: In Vivo Efficiency and Confocal Microscopy Real Time Intracellular Investigations

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

557

325. Long-Term Gene Expression after Hydrodynamic Delivery of Sleeping Beauty Transposons to Canine Liver Using Balloon Catheters

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

558

326. Efficient Microbubble- and Ultrasound-Mediated Plasmid DNA Transfer into a Specific Rat Liver Lobe Via a Targeted Injection and Acoustic Exposure Using a Novel Ultrasound System

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 205 KB

english, 2010

559

327. A Novel Prototype Device for Electroporation-Enhanced DNA Vaccine Delivery to Both Skin and Muscle

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 205 KB

english, 2010

560

328. Safety and Feasibility of Transvenous Limb Perfusion with Saline in Human Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

561

329. A Genetically Engineered Multifunctional Vector for HER2 Targeted Cancer Suicide Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 206 KB

english, 2010

562

330. Comparative Analysis of Enzyme and Pathway Engineering Strategies for Suicide Gene Therapy Applications

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 206 KB

english, 2010

563

331. Conditional and Effective Gene Knock-Down in Proangiogenic Macrophages To Disrupt Tumor Angiogenesis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

564

332. Cell Fusion between Mesenchymal Stem Cells and Cancer Cells: A Mechanism of Cancer Progression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

565

333. Next-Generation Lentiviral Cell Fate Control Gene Therapy Based on Engineered Variants of Human Deoxycytidine Kinase

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

566

334. Tumor-Selectivity of Replication-Competent Retrovirus (RCR) Vectors Regulated by Prostate-Specific Promoters and Cytotoxic Effect of Suicide Gene Therapy Mediated by RCR Vectors In Vitro

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

567

335. Translational Phase I Study in Advanced Cancer Patients of Dual-Function Autologous (FANG) Vaccine Incorporating Bifunctional shRNAfurin and GMCSF Transgene Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 244 KB

english, 2010

568

336. Efficient Gene Transfer in Quiescent Healthy and Cancer B Cells by a Measles Virus gp Pseudotyped LV

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 217 KB

english, 2010

569

337. Safely Exploiting Hematopoeitic Stem and Progenitor Cell Specific microRNAs To Target Transgene Expression to the Differentiated Progeny

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 217 KB

english, 2010

570

338. Therapeutic Fetal Hemoglobin Production in Erythroid Progeny of Normal and ß-Thalassemic Human CD34+ Cells Using Lentiviral Vectors Encoding γ-Globin or shRNA To Down Modulate the Transcriptional Repressor BCL11A

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

571

339. Potentially Therapeutic Levels of Fetal Hemoglobin in Erythroid Progeny of Normal and ß-Thalassemic CD34+ Cells Following Lentivirus-Mediated Delivery of a Zinc-Finger Transcriptional Activator Targeting the γ-Globin Gene Promoters

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

572

340. Development of an S/MAR Based Episomal Vector of a Specific Zinc-Finger Activator That Mediates Gamma-Globin Gene Activation, for the Gene Therapy of Hemoglobinopathies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 210 KB

english, 2010

573

342. Correction of Murine Hemophilia A Following Nonmyeloablative Transplantation of Hematopoietic Stem Cells Engineered To Express a Bioimproved Human FVIII Using a Safety-Augmented, B-Lymphoid Lineage-Restricted Lentiviral Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

574

344. Early Gestational Gene Transfer of IL-10 by Lentiviral Vector Can Prevent Arthritis in a Murine Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 245 KB

english, 2010

575

345. Expression Profile and Epigenetic Landscape Influence in a Cell-Specific Fashion Integration Site Selection and In Vivo Distribution of MLV-Vector in Patients

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

576

346. High-Definition Mapping of Retroviral Integration Sites Defines the Fate of Allogeneic T Cells after Donor Lymphocyte Infusion

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

577

348. Multiple Pathways Facilitate Transduction of Quiescent Human CD34+ Cells by HIV-1 Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

578

349. Targeted Lentiviral Gene Delivery to Cells Expressing c-fms or IL-2 Receptor

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 244 KB

english, 2010

579

350. Regulation of Transgene Expression In Vivo by Proteasomal Degradation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

580

352. Evaluation of Cytokine Effects on Lentiviral Transduction of Human CD34+ Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

581

353. Towards Quantitative High Throughput Clonality Screening: Redefining the Frontier of Retroviral Integration Analysis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

582

355. Packaging Heterologous Retroviral Vectors into HIV-1 Viral Particles

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

583

356. High Throughput Vector Integration Analysis with SeqMap 2.0

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

584

357. ‘Self Marker CD47 on Viral Envelopes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 382 KB

english, 2010

585

359. Fibrin-Conjugated Pseudotyped Lentivirus for Cell-Controlled and Spatially Localized Gene Delivery on Microarrayed Surfaces

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 382 KB

english, 2010

586

360. Improved Transduction Efficiency of Human Amniotic Mesenchymal Stem Cells Using Optimized Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 407 KB

english, 2010

587

361. Purification of Lentivirus Vector by Iodixanol Self-Forming Density Gradient

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

588

362. rAAV-Mediated Delivery of Micro RNA Scavengers Leads to Efficient and Stable Knock-Down of Cognate Micro RNAs, Upregulation of Their Natural Target Genes and Phenotypic Changes in Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

589

363. Activation of the Non-Canonical NF-kB Pathway by AAV2 Vectors: Implications in Immune Response and Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 335 KB

english, 2010

590

365. The Rous Sarcoma Virus Promoter Carried by a rAAV Vector Is Resistant to DNA Methylation in Primate Muscle and Liver

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 358 KB

english, 2010

591

366. The Hybrid AAV2.5 Vector Transduces Neurons Bordering the Third and Fourth Ventricles after Lateral Ventricular Infusion in Rats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 194 KB

english, 2010

592

367. Differential Lymphatic Transport of Chimeric and Naturally Occurring AAV Strains

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 194 KB

english, 2010

593

368. Optimizing Liver Transduction of the Low Seroprevalent AAV rh32.33

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 461 KB

english, 2010

594

369. Role of a Cellular Chaperone Protein, FKBP52, in Uncoating of AAV2 Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 444 KB

english, 2010

595

371. Optimizing Capsid Composition for the Production of Recombinant Adeno-Associated Virus Using the Baculovirus Expression System

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 325 KB

english, 2010

596

372. Genetically Re-Engineered AAV Rep78 Identifies Sequence-Restricted Inhibitory Regions Affecting Adenoviral Replication

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 490 KB

english, 2010

597

375. rAAV1 Infect Mouse Bone Marrow-Derived Dendritic Cells through Receptors with α2,3 and α2,6 Sialic Acids

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 291 KB

english, 2010

598

376. Using AAV Vector as Carrier for Biosensor: Detecting microRNA's Activities by Reverse Infection of AAV Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 291 KB

english, 2010

599

377. Clearance of Herpes Simplex Virus by Orthogonal Manufacturing Process of Recombinant Adeno-Associated Virus (rAAV) Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 310 KB

english, 2010

600

378. Production of a Novel Class of Human Polyomavirus JC Vectors Using Baculovirus Expression System

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

601

379. Induction of Cell Surface Integrins in Lymphomas and Potential Applications to Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

602

380. FX-Binding Ablated Ad5 Do Not Transduce Hepatocytes but Exhibit Splenic Uptake at High Doses

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 213 KB

english, 2010

603

381. Targeting Adenovirus Cell Entry Via Tumor-Associated EphA2 by Insertion of a Ligand Peptide into Different Loops of the Short Fiber of Ad41

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 213 KB

english, 2010

604

382. Induction of a Prostate Specific Promoter and Replication Kinetics by p21/Waf-1 shRNA Armed CRAd

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 213 KB

english, 2010

605

384. Inhibition of PD-1 Greatly Augments Therapeutic Effect of Suicide Gene Therapy for Gastrointestinal Cancers

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 213 KB

english, 2010

606

385. Method for Fast and Easy Adoption of First-Generation Adenoviral Vectors to High-Capacity Adenoviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 213 KB

english, 2010

607

386. PAUF Targeting RNA-Based Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 272 KB

english, 2010

608

387. Combining Oncolytic Adenoviruses and Antibody Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

609

388. Direct Injection of HDAd into the Liver Is as Effective but Less Toxic Than Intravenous Injection for Crigler-Najjar Syndrome Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

610

390. Optimized Adenovirus Targeting through Virus-Displayed Peptide Library Screening in a Prostate Cancer Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

611

392. Macropinocytosis Is the Major Pathway Leading to Transfection for a Charge-Reversal Amphiphile

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

612

393. Ornithine-Based Cationic Lipids: Highly Charged Dendritic Headgroups, New Lipoplex Phases and Degradable Lipids

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

613

394. Nanoparticle-Mediated Delivery of Genes and Imaging Agents to the Brain

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

614

397. Dual Function Effect on PEI-Mediated Gene Delivery by Diphtheria Toxin-Derived Ligand and Fusogenic Domains

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 264 KB

english, 2010

615

400. Histidine-Rich Amphipathic Peptides Promote Efficient Delivery of Nucleic Acids into Mammalian Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

616

402. Evaluation of Polymer-DNA Nanoparticles for Gene Transfer to Cardiac Muscles

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

617

403. Chemical Manipulation of AAV Tissue Tropism

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 302 KB

english, 2010

618

404. Versatile Self-Assembling Nanoparticles for Enhanced siRNA Delivery into Mammalian Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 276 KB

english, 2010

619

405. Novel L-Lysine Reducible Copolymers for Efficient Nonviral Gene Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 490 KB

english, 2010

620

406. JL1-Directed Small Interfering RNA Delivery Using Anti-JL1 Immuno-Sendai Viroplexes to T-Leukemic Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 433 KB

english, 2010

621

407. Antibody-Directed Drug Delivery Using Protein A-Based Carriers

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 433 KB

english, 2010

622

409. Creation and Rescue of a Severe Hyperammonaemic Phenotype in Ornithine Transcarbamylase Deficient spfash Mice Using rAAV and shRNA

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

623

410. Long Term Correction of Biochemical and Neurological Abnormalities of Metachromatic Leukodystrophy Model Mice by Systemic Neonatal Injection of Serotype 9 AAV Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

624

411. Neural Stem Cells as a Therapeutic for Krabbe's Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

625

413. Widespread Enzymatic Correction of CNS Tissues by a Single Intracerebral Injection of Therapeutic Lentiviral Vector in Leukodystrophy Mouse Models

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 294 KB

english, 2010

626

415. Towards Targeted Transgene Integration in Human Hematopoietic and Induced Pluripotent Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 263 KB

english, 2010

627

416. Optimization of the Expression Cassette Using an AAV2/8 Based Vector for the Treatment of Ornithine Transcarbamylase Deficiency

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 244 KB

english, 2010

628

417. Generation of Early AMD Mouse Model by Induction of Oxidative Stress

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 244 KB

english, 2010

629

418. Increased Expression of Human Glucocerebrosidase Activity in Bone Marrow of Gaucher Mice Following Macrophage Mediated Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

630

419. Cell Sheet Engineering toward Bioengineering of Functional Neo-Islets

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

631

420. Liver-Specific Over-Expression of PGC-1α Does Not Induce a Diabetic Phenotype

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

632

421. Promoter Selection for AAV Vector-Mediated Factor IX Expression in Skeletal Muscle

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

633

423. AAV-Mediated Intramyocardial VEGF-B167 Expression Exerts a Potent Cardioprotective Effect in Small and Large Animal Models of Myocardial Infarction and Heart Failure

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 343 KB

english, 2010

634

424. Osteopontin Enhances the Angiogenic Potential of Endothelial Progenitor Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 343 KB

english, 2010

635

425. Arginine and Tetrahydrobiopterin Synergistically Potentiate the Antirestenotic Effect of Vascular Gene Therapy with Inducible Nitric Oxide Synthase

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

636

426. Intra-Myocardial Administration of ACRX-100 Promotes Cardiac Benefit and Improves Angiogenesis in a Porcine Model of Heart Failure

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

637

428. Systemic Delivery of Interleukin-10 by an AAV Vector Attenuates Obesity-Related Glomerulopathy in Zucker Fatty Rats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 620 KB

english, 2010

638

429. AAV/B7H1-Gene Delivery Lowers Th1 Cytotoxic T Lymphocyte Response In Vitro and Atherogenesis In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 620 KB

english, 2010

639

430. Dose-Dependent Angiogenesis by Human and Murine VEGF Is Species-Specific

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

640

431. AAV-Sema3A-Mediated Recruitment of Neuropilin-1-Expressing Mononuclear Cells (NEMs) Contributes to Vessel Stabilization and Normalization

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

641

432. Identification of AAV6 as Most Efficient Vector for Transvascular Gene Transfer into Porcine Myocardium Based on an In Vitro Model for Prediction of the Cardiac Gene Transfer Performance

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 283 KB

english, 2010

642

433. Percutaneous Transendocardial Adminstration of AAV6 and AAV8 Vectors to Rhesus Macaques Results in Highly Efficient and Global Cardiac Gene Transfer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 235 KB

english, 2010

643

434. Gene Transfer of VEGF165, HGF, Urokinase and Their Combinations Using Novel PC4W Plasmid for Induction of Vasculogenesis in Mice Hind Limb Ischemia Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 536 KB

english, 2010

644

435. PEGylation of Helper-Dependent Adenoviral Vector-Mediated Long-Term Expression of Human Apolipoprotein A-I Reduces Atherosclerosis in LDLR Deficient Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 518 KB

english, 2010

645

436. AAV/IL13-Gene Delivery Lowers Atherogenesis in LDLR KO Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 518 KB

english, 2010

646

437. AAV/hSTAT3-Gene Delivery Lowers Atherosclerosis in LDLR KO Mice on High Cholesterol Diet

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 597 KB

english, 2010

647

438. Intramuscular Delivery of ACRX-100 Enhances Angiogenesis in Rabbits with Hind Limb Ischemia

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 597 KB

english, 2010

648

439. A Comparison of Three Liposomal Formulations for Gene Delivery from Coronary Artery Stents

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 730 KB

english, 2010

649

440. Efficient and Localized Gene Transference in Rat Heart by Electroporation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 353 KB

english, 2010

650

441. Gene Delivery of Erythropoietin Protects Retinal Ganglion Cells in DBA/2J Glaucomatous Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 353 KB

english, 2010

651

442. Pan-Retinal Secretion of GDNF Following Intravitreal Transduction of Müller Glia Slows Photoreceptor Loss in Retinal Degeneration

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 381 KB

english, 2010

652

443. AAV-Mediated Gene Supply of Non-Erythropoietic Erythropoietin Derivatives Improves Photoreceptor Morphology and Function in Models of Retinal Degenerative Diseases

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

653

444. Early Indicators of Biological Efficacy in a Phase 1/2 Gene Therapy Trial for Leber Congenital Amaurosis (LCA)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

654

445. Positive Responses in a Mouse Model of Leber Congenital Amaurosis after Treatment with Compacted DNA Nanoparticles

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

655

446. Adeno-Associated Virus Mediated Gene Therapy with a Rhodopsin Ribozyme for Treatment of Autosomal Dominant Retinitis Pigmentosa (Adrp)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

656

447. POD DNA Nanoparticles Rescue an Adult Mouse Model of Retinal Degeneration

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

657

448. Protection Against Laser-Induced Choroidal Neovascularization Following Delivery of an Adenovirus Expressing a Soluble Form of Human CD59 to Murine Retina

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 287 KB

english, 2010

658

449. Dose Finding Study in Galactosialidosis Mice Using scAAV2/8-LP1-ppca Virus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

659

450. Characterization of 12 AAV Vectors for Intravascular Delivery To Target CNS and Detarget Non-CNS Tissues by miRNA Regulation: Implications in Treatment of Canavan Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

660

451. Efficient Expression of Smn from Integration-Deficient Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

661

453. Genetically Engineered Suppressor Cells Target CNS and Inhibit Activated Effector T-Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 309 KB

english, 2010

662

454. First Generation AAVmicroutrophin Vector Infused into the Isolated Pelvic Limb of a Canine Model for Duchenne Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 281 KB

english, 2010

663

455. Characterization of A20 as an Inhibitor of NF-κB Activation in Dystrophic Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 281 KB

english, 2010

664

456. Plasticity of Skeletal Muscle Cells during Muscle Injury and Repair – A Dedifferentiation Study

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 301 KB

english, 2010

665

458. External, Non-Invasive Monitoring of Progressive Cardiorespiratory Dysfunction in a Canine Model of DMD

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 309 KB

english, 2010

666

459. The Polyproline Site in Hinge 2 Influences the Functional Capacity of Truncated Dystrophins

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 262 KB

english, 2010

667

460. Combining Gene and Stem Cell Therapy in the Treatment of Dysferlinophaties

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 262 KB

english, 2010

668

461. Deletion of Functional Domains of Dp116 Leads to Altered Sarcolemmal Expression and Subcellular Localization When Expressed in Skeletal Muscle

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 282 KB

english, 2010

669

462. A New Therapeutic Approach for Duchenne Muscular Dystrophy: Restoration of the Dystrophin Reading Frame with a Meganuclease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 262 KB

english, 2010

670

463. Regulatory Cassettes Derived from the Slow Troponin I Gene Confer High-Level Expression in Skeletal Muscle after Gene Transfer Mediated by Helper-Dependent Adenovirus and Recombinant AAV

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 262 KB

english, 2010

671

464. Cell Therapy of Muscular Dystrophy with Engineered Cd133+ Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 262 KB

english, 2010

672

465. Improved Regenerative Capacity of Adult Stem Cells Isolated from Injured Skeletal Muscle of Mouse

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 289 KB

english, 2010

673

466. Sarcolemmal nNOS Anchoring Reveals a Functional Difference between Dystrophin and Utrophin

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

674

467. Detailed Physical Mapping of Deletion Breakpoints in the GSHP Dog Model for DMD

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

675

468. Lentiviral Vector Mediated Delivery of Full-Length Dystrophin for Gene Therapy of Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 280 KB

english, 2010

676

469. Elimination of Cancer Stem Cells with Genetically Engineered T Cells Expressing a Stage Specific Embryonic Antigen-1 (SSEA1) Chimeric Antigen Receptor

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

677

470. Targeting Cancer Stem Cells in Osteosarcoma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

678

471. CMV-Specific HER2-Redirected T Cells for the Adoptive Immunotherapy of Glioblastoma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

679

473. Evaluating the Combination of 5’Azacytidine and MAGE-A4-Specific T Cell Therapy as a Potential Immunotherapeutic Strategy for Refractory Hodgkin's Lymphoma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 261 KB

english, 2010

680

474. The iCD40.MyD88 Dendritic Cell (DC) Gene Switch Leads to Potent Anti-Tumor Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

681

476. TCR Engineered and In Vitro Expanded T Lymphocytes Recapitulate the Properties of Central Memory T Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 255 KB

english, 2010

682

477. Phase I Safety and Immunogenicity of HPV 16 and 18 DNA Vaccines Delivered Via Electroporation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 255 KB

english, 2010

683

478. Improving Adoptive T Cell Therapy by Transduction of TIL with a Vector That Mediates the Inducible Expression of IL-12 in the Tumor Microenvironment

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 433 KB

english, 2010

684

479. Robust and Sustained Ex Vivo Expansion of NK Cells with Artificial APCs Bearing Membrane-Bound IL-21 Is Associated with a Distinct Phenotype and Transcriptional Profile

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 417 KB

english, 2010

685

480. STAT3-Inactivated Dendritic Cell Vaccination Could Induce Stronger Anti-Tumor Immune Responses

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

686

482. Bid Is a Key Factor for Eliciting the Antiproliferative Activity of IFN-aL Mediated by TRAIL

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

687

483. Oral Adeno-Associated Virus-neu (AAV-neu) Immunotherapy Inhibits the Growth of neu+ Murine Breast Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

688

484. Tumor-Specific Antitumor Activity of mIL-10 Revealed by Hydrodynamic Tail Vein Injection of pCMV-mIL Plasmids in Tumor-Bearing Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

689

485. Liver Specific, Inducible, AAV-Mediated IL12 Delivered Resulted in a Strong Anti Tumoral Inmune Response in a Murine Model of Liver Metastasis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

690

487. Xenograft Expanded FANG Autologous Tumor Cell Vaccine Development and Manufacturing for Clinical Use

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 279 KB

english, 2010

691

488. Optimization of Measles Virus Infected Mesenchymal Stem Cell Carriers for Clinical Testing in Patients with Recurrent Ovarian Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

692

489. Expression of Interferon Alpha from an Oncolytic Adenovirus Vector Decreases Toxicity but Retains the Anti-Tumor Efficacy of the Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

693

490. Measles Virus Encoding H. pylori Neutrophil-Activating Protein for Treatment of Metastatic Breast Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 285 KB

english, 2010

694

491. Carrier Cells Delivery of Inducible Oncolytic Adenovurs for Brain Tumor

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 235 KB

english, 2010

695

492. Optimizing the Genomic Stability, Transgene Expression, and Therapeutic Efficacy of Replication-Competent Retrovirus Vectors for Prodrug Activator Gene Transfer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 235 KB

english, 2010

696

493. Gene Therapy for Head and Neck Squamous Carcinoma Cells by IAI.3B Promoter-Driven Oncolytic Adenovirus-Infected Carrier Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 235 KB

english, 2010

697

494. Evaluation of Monocytes and Cancer Cells as Carriers for Oncolytic Adenoviruses in Immune-Competent Models of Pancreatic Cancer in Syrian Hamsters

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 235 KB

english, 2010

698

495. Combination Therapy for Breast Cancer Using Oncolytic Viruses and Histone Deacetylase Inhibitors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 284 KB

english, 2010

699

496. Combination Oncolytic Virotherapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

700

497. Conditionally Replicative Adenovirus with an Adenoviral Major Late Promoter-Driven Imaging Cassette Predicts In Vivo Anti-Tumor Effect

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

701

498. Susceptibility of Neuroblastoma Primary Tumor-Initiating Cells to Oncolytic Herpes Simplex Virus Is Determined by Nectin-1 Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

702

500. Replication-Competent Retrovirus Vector-Mediated Suicide Gene Therapy Achieves Significant Therapeutic Efficacy Against Human Malignant Mesothelioma Xenografts

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 280 KB

english, 2010

703

502. Silencing a p53 Inhibitor in Cancer Cells Increases Killing Potency of Oncolytic Adenovirus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

704

504. Characterization of a Novel Dual-Lock Oncolytic Cadvex-Vector That Combines Efficient Prostate-Specific Replication with Cytokine Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

705

505. A Probasin Promoter, Conditionally Replicating Adenovirus that Expresses the Sodium Iodide Symporter (NIS) for Radiovirotherapy of Prostate Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

706

506. Adenovirus Vectors Transductionally Retargeted to Her2/neu for Prostate Tumour Treatment

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 263 KB

english, 2010

707

507. The Potential of Conditionally Replicating Adenovirus Expressing TIMP2 for Ovarian Cancer Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 196 KB

english, 2010

708

509. Truncated CD19 Fused with Mutant Human Tmpk: A Versatile Cell Fate Control Safety Cassette

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 215 KB

english, 2010

709

510. Muscle Directed Systemic Cancer Gene Therapy for Lymphoma Using Type 8 AAV Vector Expressing mda-7/IL24

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 196 KB

english, 2010

710

511. A Liposome Formulation with High Accumulation in Tumor Tissue for Targeted Delivery of Nucleic Acid

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 453 KB

english, 2010

711

512. 15-LO-1 Induced Apoptosis Inhibits Tumour Growth and Extends Survival in Rat Glioma Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 433 KB

english, 2010

712

513. An Efficient Targeted Gene Therapy Using Brain Tumor-Specific Promoter

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 433 KB

english, 2010

713

514. Nitric Oxide Synthase One Homologous Recombinant Negative (NOS1 -/-) Mice Are Protected from Thoracic Irradiation Induced Seizures by Esophageal Administration of Manganese Superoxide Dismutase Plasmid Liposome Complex (MnSOD-PL)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 206 KB

english, 2010

714

515. Maternal Manganese Superoxide Dismutase Plasmid/Liposome Complex (MnSOD-PL) Gene Therapy of E13 Pregnant Mice Reduces Total Body Irradiation Induced Growth Retardation in Newborn Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 206 KB

english, 2010

715

516. Adenovirus-Relaxin Gene Therapy on Keloids: Attenuated Proliferative Response and Collagen Degradation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 231 KB

english, 2010

716

517. Inducing Systemic Anti-Tumor Immune Responses through Direct Tumor Targeting

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

717

518. Angiotensin Type 2 Receptor Mediated Apoptosis of Human Prostate Cancer Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

718

519. Abstract Withdrawn

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

719

521. Lentiviruses Containing a Modified Ankyrin-1 Promoter Direct Uniform, Copy-Number-Dependent Expression of a γ-Globin Gene at Therapeutic Levels

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

720

522. Non-Myeloablative Conditioning Regimen To Increase Engraftment of Gene-Modified Hematopoietic Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

721

523. In Vivo Selection of Hematopoietic Stem Cells by Expressing a Truncated Epo Receptor (tEpoR)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 219 KB

english, 2010

722

525. Diminished End Organ Damage and Correction of Anemia in Sickle Cell Mice Treated with Non-Myeloablative Busulfan Conditioning and Y-Globin Lentiviral Vector Transduced HSCs

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

723

526. Small but Powerful, Predicting the Outcome of Gene Therapy Trial Using NSG Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

724

528. Transgenic Expression of Cytidine Deaminase (CDD) for Myeloprotective Purposes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

725

529. Development of a Pre-Clinical Model for the Gene Therapy Treatment of Wiskott-Aldrich Syndrome (WAS)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

726

530. Lentiviral Gene Therapy of Murine Hematopoietic Stem Cells Using Codon Optimized IL2RG cDNA: A Comparison of Multiple Promoter Elements and Transplant Conditions

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 249 KB

english, 2010

727

531. Human Fetal Liver HSCs Demonstrate High Transduction Rates and Sustained Transgene Expression Following Implantation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

728

532. FANG Autologous Tumor Cell Vaccine Development and Manufacturing

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 265 KB

english, 2010

729

534. A Combination of Codon Optimization and Amino Acid Substitution in the Human Factor IX Gene Yields a High Level of Superactive Factor IX

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

730

536. Phase I Safety and Pharmacokinetic Study of Systemic p53 Gene Therapy (SGT-53) Using Immunolipoplexes Targeted by Anti-Transferrin Receptor (TfR) Single Chain Antibody Fragment (scFv): Updated Results

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 309 KB

english, 2010

731

537. Induction of Type I Interferon (IFN) by Adenovirus-Encoded Small RNAs

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 363 KB

english, 2010

732

538. Immunological Effects of Oral and Intravenous Cyclophosphamide in Cancer Patients Treated with Oncolytic Adenoviruses

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

733

539. The Role of TGF-β and IL-10 in the Development of Immune Tolerance Following Hepatic Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 254 KB

english, 2010

734

540. Murine Hepatocytes Transduced with Y-F Mutant Capsid Are More Resistant to Killing by Capsid Specific CD8+ T Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

735

541. Abstract Withdrawn

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

736

544. Robust and Sustained Factor IX Expression by IV Administration of AAV8-Based Vectors in Macaques

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

737

545. Induction of Transgene Immunological Tolerance by a Single Injection of miR-142-Regulated Integrase Defective Lentiviral Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 279 KB

english, 2010

738

546. T Cell Hyperactivity in Cmah Null Mice: A Pre-Clinical rAAV Immunotoxicity Model?

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

739

547. Complement-Mediated Clearance of AAV by Tissue Resident Macrophages Limits Transduction of Target Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 286 KB

english, 2010

740

548. Overcoming Anti-AAV Neutralizing Antibodies with Alternate Serotype and Pharmacological Strategies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

741

549. Switching the Fiber Knob of Oncolytic Adenoviruses To Avoid Neutralizing Antibodies in Human Cancer Patients

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

742

550. Depletion of B Cells by Anti-CD20 Partially Regulates Anti-FVIII Antibody Production in the Nonviral Gene Therapy Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

743

551. Sheep Harbor Naturally-Occurring Antibodies to Human AAV: A New Large Animal Model for AAV Immunology

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

744

552. Effects of Various TLR/Inflammation Pathways on Humoral and Cellular Immune Response to rAAV1 in Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

745

553. Monitoring and Suppression of Immune Responses to AAV-Mediated Gene Therapy in Canine Muscle

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 234 KB

english, 2010

746

554. Prevalence of Neutralizing Antibodies Against Adeno-Associated Virus 2, 8 and 9 in Non-Human Primate Colonies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

747

555. Dual Function BAFF Receptor Aptamers Block BAFF1 Ligand Induced Proliferation and Deliver siRNAS to NHL Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

748

556. Efficient Delivery of siRNAs into Primary Cells In Vitro and Systemically In Vivo Using Chemically Modified PepFect Peptides

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

749

557. Reduction of Mutant Huntingtin by Antisense Oligonucleotides Ameliorates Phenotypic Deficits in YAC128 Mouse Model of Huntington's Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

750

558. Antibody-Mediated Targeting of pH-Responsive Nanoparticles Facilitates siRNA Delivery to Cancer Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 262 KB

english, 2010

751

559. Adenoviral shRNA-Mediated Knockdown of TNFR1(P55) Either Locally in the Knee Joint or Systemically Ameliorates Collagen-Induced Arthritis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

752

560. Specific and Efficacious RNA Aptamer Against Hepatic Metastasis of Colon Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

753

561. Development of Aptamer Therapeutics Against Cell Surface Receptors for Lymphoma and Auto-Immune Diseases

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 386 KB

english, 2010

754

562. A Microscopic Study of the Intracellular Fate of Cationic Mediated siRNA Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 368 KB

english, 2010

755

563. Deriving Effective Anti-HIV Guide Sequences from Enhanced Primary microRNA Secondary Structures

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 396 KB

english, 2010

756

564. “Bifunctional” shRNA Strategy for Cancer Gene and Cell Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

757

565. Feasibility and Effectiveness of Exon 51 skipping in Human-Like mdx Mutation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

758

566. The Effect of RNA Interfering α1,3GT and NF-kB in Cardiac Xenotransplantation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

759

567. Inhibition of Breast Cancer Cell Growth Via Silencing IKKe

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

760

568. Cationic Polymer Mediated siRNA Delivery for P-glycoprotein (P-gp) Down-Regulation in Tumor Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 444 KB

english, 2010

761

569. Gene Editing Induces Double-Stranded Breakage in Mammalian Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 406 KB

english, 2010

762

571. Production of a rAAV1-CB-hAAT Human Clinical Trial Lot by rHSV Co-Infection of Suspension BHK Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 288 KB

english, 2010

763

572. RNA Based Plasmid Selection System for Antibiotic-Free Plasmid DNA Vector Production

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 288 KB

english, 2010

764

573. Robust Recombinase-Free System Production of Helper-Dependent Adenoviral Vectors with Negligible Contamination by Delayed Packaging Process

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 821 KB

english, 2010

765

574. Removal of Clinically Relevant Contaminants from cGMP Grade Preparations of Plasmid DNA: Implications for Non-Viral Gene Therapy and DNA-Based Vaccines

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 735 KB

english, 2010

766

575. Escherichia coli Plasmid DNA Fermentation Strain and Process-Specific Effects on Vector Yield, Quality and Transgene Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 735 KB

english, 2010

767

577. Counting and Sizing of Viral Vector Particles and Aggregates by Nanoparticle Tracking Analysis (NTA)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 361 KB

english, 2010

768

578. Manufacturing and Stability Testing of a GNE-Lipoplex for a Clinical Trial for the Treatment of Hereditary Inclusion Body Myopathy Type (HIBM)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 386 KB

english, 2010

769

579. Dissolved Oxygen and pH Profile Evolution in Shake Flasks during 293 Cell Growth and Infection with Adenovirus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

770

581. Transient Expression of a Therapeutic Dystrophin Transgene in Duchenne Muscular Dystrophy Revealed by T Cell Mediated Immunity

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

771

582. Induction of Oral Immunotolerance to rAAV9-Microdystrophin in Canine X-Linked Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

772

583. Baculovirus-Engineered Mesenchymal Stem Cells Heal the Critical-Sized Femoral Segmental Bone Defects in Rabbits

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 234 KB

english, 2010

773

584. Genetic Evidence for a Structural Role of α-dystrobrevin-3 within the Dystrophin-Glycoprotein Complex

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 234 KB

english, 2010

774

585. rAAV8-Mediated Protein-Anchoring Therapy for Targeting Collagen Q-Tailed Acetylcholinesterase to the Neuromuscular Junction

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 650 KB

english, 2010

775

586. A Single Patient, Multiple Intravenous (IV) Infusion, Clinical Trial of GNE-Lipoplex for Therapy of Hereditary Inclusion Body Myopathy (HIBM)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 636 KB

english, 2010

776

587. Successful Expression of a R16/17 Micro-Dystrophin Gene in Dystrophic Dogs with a Tyrosine-Mutant AAV Vector and a Brief Course of Immune Suppression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

777

588. Transcription Factor Binding by Plasmid DNA Is Needed for Interaction with Microtubules and Trafficking in Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

778

589. Quantitative Analysis of the Effect of Functional Peptides on Intracellular Trafficking of Polymer Gene Delivery Systems

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

779

590. The Effects of PNA-Based prePEGylation on the In Vitro and In Vivo Properties of DNA Polyplexes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

780

591. Particle Tracking for Intracellular Trafficking of Octaarginine-Modified Liposome: Comparative Study with Adenovirus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 225 KB

english, 2010

781

592. Advanced In Vitro Models Combined with Mathematical Representations Yield New Quantitative Understanding of Synthetic Gene Delivery System Performance

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 333 KB

english, 2010

782

593. Gene Therapy of the Blood Brain Barrier: Systemic Delivery of shRNA-Producing Plasmid DNA for Transient Suppression of MRP4 Drug Efflux Transporter Protein

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 288 KB

english, 2010

783

594. PEGylated Polyacridine-Peptide DNA Open-Polyplexes with Long Pharmacokinetic Half-Life Have Improved Hydrodynamic Stimulated Gene Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 455 KB

english, 2010

784

595. Successful Gene Delivery with Receptor-Targeted PAMAM Dendrimers Occurs Via Caveolae-Mediated Pathways

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 387 KB

english, 2010

785

596. Systemic Delivery of AAV2/9 Vectors Improve Cardiac Function in Pompe Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 387 KB

english, 2010

786

597. Coordinated Co-Expression of VEGF164 and PDGF-BB Prevents Aberrant Angiogenesis and Accelerates Vascular Stabilization

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 550 KB

english, 2010

787

598. Mitral Valve Disease Is Due to Abnormal Collagen Structure in Canine Mucopolysaccharidosis I and VII and Can Be Reduced with Neonatal Intravenous Injection of a Gamma Retroviral Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 345 KB

english, 2010

788

599. AAV9-Mediated proBNP Delivery Improves Diastolic Performance in a Model of Spontaneous Hypertensive Rats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 365 KB

english, 2010

789

600. In Vivo Over-Expression of a β-Adrenergic Receptor Kinase Inhibitor Using scAAV6-Mediated Molecular Cardiac Surgery with Recirculating Delivery (MCARD™) Enhances Cardiac Function

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

790

601. AAV-Mediated SERCA2a Expression Improves the Electrocardiographic Profile of Aged mdx Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

791

603. SDF-1α-Engineered Cell-Based Therapy for Diabetic Wound Healing

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

792

604. High Resolution Analysis of the Intratumoral Spread of an Intravenously Administered Oncolytic Virus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 249 KB

english, 2010

793

605. ABSTRACT WITHDRAWN

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 179 KB

english, 2010

794

606. Combination of Anti Angiogenic Cancer Therapy with Systemic Oncolytic Virotherapy To Treat Established Tumors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 250 KB

english, 2010

795

609. Improving the Lytic Capacity of a Conditionally Replicative Adenoviruses by Incorporating Promoter Elements Responsive to Tumor Environmental Conditons

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

796

610. Defining the Role of Innate Immune Mechanisms in the Efficacy of Oncolytic VSV Virotherapy in a Fully Immune Competent Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

797

611. Differential Effects of VSV and Cyclophosphamide on NK Cells Leads to Antagonistic Effects on Anti-Tumor Therapy of a Murine Mesothelioma Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

798

613. Sustained Partial Phenotypic Correction of Hemophilia B in the Dog by a microRNA-Regulated Lentiviral Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 198 KB

english, 2010

799

614. Timing of Anti-T Cell Immunosuppressive Therapy Is Critical for the Safety of Liver Mediated AAV2 Transduction in Rhesus Macaques

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

800

615. Factor IX-R338L (FIX Padua) as a Novel Alternative for the Treatment of Canine Severe Hemophilia B

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 198 KB

english, 2010

801

616. Erythropoeisis Sustained 1 Year by the EPODURE BioPump in Patients with Chronic Kidney Disease: Further Results of Phase I/II Proof of Concept Trial

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 198 KB

english, 2010

802

617. Lentiviral Gene Therapy of RAG1 Severe Combined Immunodeficiency: Risks and Implications for Treatment

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 241 KB

english, 2010

803

618. Disruption of Glucocorticoid Regulation in Therapeutic Human T Cells with Zinc Finger Nucleases

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 241 KB

english, 2010

804

619. Lentiviral Gene Transfer under Optimized Cytokine Conditions Facilitates Polyclonal Hematopoiesis of Recipient Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 261 KB

english, 2010

805

621. Persistent Long Term Lentiviral Correction of the CFTR Gene Defect in Cystic Fibrosis Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 287 KB

english, 2010

806

622. Development of Optimized hCFTR Gene Sequence and Prolonged Expression Vector for Cystic Fibrosis Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

807

623. Expression of Human Alpha-1 Antitrypsin in Mice and Dogs Following AAV6 Vector-Mediated Gene Transfer to the Lungs

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

808

624. Engineered Zinc Finger Transcription Factors for the Treatment of Gain of Function Mutations

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

809

625. AAV Vectors for Tissue-Specific, Regulated Expression in Heart, Liver and Lung

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

810

626. Repairing Huntingtin RNA by Trans -Splicing

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

811

627. Towards Personalized Arthritis Gene Therapy: Screening of Computationally-Designed Promoters in an In Vivo Mouse Model and in Human Synovial Fibroblasts

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 295 KB

english, 2010

812

628. Contribution of PDGFRα-Positive Bone Marrow Cells for Epithelial Regeneration in Genetic Blistering Skin Disease, RDEB

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

813

629. Adult Muscle-Derived Stem Cells Rescue Progeria Stem Cell Dysfunction and Promote Angiogenesis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

814

630. AAV-2 Delivered Factor IX Is Secreted with Full Clotting Activity from Human Adipose Stromal Cells Differentiated towards Hepatocytes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

815

631. Nonhuman Primate Cord Blood Expansion and Transplantation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

816

632. Stealth Delivery: Combining Site-Specific Integration and Cassette Design To Achieve Robust Expression without Impacting Endogenous Gene Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 265 KB

english, 2010

817

633. Generation of Insulin-Secreting Cells from Human Induced Pluripotent Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

818

634. Simple and Efficient Preparation of Immaculate Human iPS Cells with Transgene-Free and No Chromosomal Damages Using an Extranuclear Manipulation System of Genetic Information, PlasmExTM

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

819

635. Myogenic Potential Enhancement of Muscle Stem Cell by Reducing NF-kB Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

820

636. Mapping of Transposon Integration Sites in Murine and Canine Liver after Transposition from a High-Capacity Adenoviral Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

821

637. Endogenous miRNA-Regulation Can Be Used To Enhance Transgene Product-Directed Immune Responses Induced by Adenovirus-Based Genetic Vaccines

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 406 KB

english, 2010

822

639. Sustained Therapeutic hFIX Levels in Rhesus Macaques without Neutralizing Anti-hFIX Antibody Development Following Catheter-Based Delivery of HDAd

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

823

640. Fully Retargeted HSV-1 Infection Directed by Re-Engineered Glycoprotein D (gD) Is Augmented by Hyperactive gB Mutations

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

824

641. Gene Expression Profiling of Adenovirus 5 Infections: Identifying Key Pathways That Affect Oncolytic Adenovirus Replication

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 294 KB

english, 2010

825

642. The Cytoplasmic Sensor, NOD2, Induces the Innate Immune Response and Transgene Silencing of Vector after Systemic Administration of Helper-Dependent Adenovirus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 267 KB

english, 2010

826

644. Chim3 as a New Transgene for Anti-HIV-1/ AIDS Gene Therapy: Antiviral Effect and Survival Advantage Features in a Preclinical In Vitro Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 288 KB

english, 2010

827

645. In Vivo Selection of CD4+ T Cells Transduced with a Gamma-Retroviral Vector Expressing a Single-Chain Intrabody Directed Against HIV-1 Tat

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 269 KB

english, 2010

828

646. Safety and Efficacy of Autologous CD4+ T cells Transduced with a Lentiviral Vector Delivering Anti-HIV RNA Antisense env in HIV+ Subjects Failing One or More HAART Regimens

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 298 KB

english, 2010

829

647. Lentiviral Delivery of TRIM-Cyclophilin Restriction Factors Against HIV-1

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

830

648. DNA Vaccination with a Consensus H1HA Influenza Immunogen Induces Immune Responses Capable of Protecting from a Lethal Novel A/H1N1-2009 and A/H1N1-1918 Challenge

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

831

649. Induction of Immune Responses Against Multiple Species of Ebolavirus Using DNA and Retroviral-Based Virus-Like Particles Bearing the Envelope Glycoprotein of a Single Species Zaire Ebolavirus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

832

651. Overexpression of the SLAM Receptor Adaptor Molecule EAT-2 as a Novel Strategy for Enhancing Cellular Immunity to HIVantigens

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 440 KB

english, 2010

833

652. A Powerful Retroviral Vector for TCR Gene Therapy: Silencing of Endogenous TCR Improved Expression and Anti-Tumor Reactivity of Transduced Tumor-Specific TCRs in Human Lymphocytes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 394 KB

english, 2010

834

653. Organ Specific Oncolytic Virus Incorporated Antigen Libraries (OSOVIAL) for the Immunotherapy of Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 411 KB

english, 2010

835

654. Editing Human Lymphocyte Specificity for Safe and Effective Adoptive Immunotherapy of Leukemia

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

836

655. Constitutive 4-1BBL Expression Dramatically Increases Proliferation, Survival and In Vivo Persistence of CD19-Targeted T Cells, Promoting Complete Tumor Elimination

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

837

656. An A20-Silenced, Stroma-Targeted DC Vaccine Reverses the Immunosuppressive Tumor Microenviroment Resulting in Potent Antitumor Activity

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

838

657. A CD46-Targeting Recombinant Protein that Increases the Therapeutic Efficacy of Monoclonal Antibodies for Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

839

659. Effective Dendritic Cell Immuno-Gene Therapy of Established ErbB2-Expressing Tumors Using Pentostatin-Based Host Immune Depletion and Subsequent Host Th1/Tc1 Cell Transfer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

840

660. Alleviation of Potentially Adverse shRNA Off-Targeting Via AAV Vector-Encoded Passenger Strand Decoys

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

841

661. Multiplexing of snoRNAs for HIV-1 Inhibition

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

842

662. Delivery of Chemo-Sensitizing siRNAs to HER2-Positive Breast Cancer Cells Using RNA Aptamers

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

843

663. Methods for Isolating Cell-Internalizing RNA Aptamers for Targeted siRNA Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

844

664. Treatment with the TLR9 Agonist MGN1703 in Patients with Metastatic Solid Tumors -EFFICACY Results of a Phase 1 Clinical Study

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

845

665. miR-7 Inhibits Schwannoma Tumor Growth by Directly Targeting p21-Activated Kinase 1 and Epidermal Growth Factor Receptor

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

846

666. Nanoparticles are Effective Vehicles for Systemic Delivery of 2’OMePS Antisense Oligonucleotides in Exon Skipping-Mediated Dystrophin Restoration

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 327 KB

english, 2010

847

667. Therapy for Vaso-Occlusion in Sickle Cell Disease Includes RNA Aptamer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 529 KB

english, 2010

848

668. GMP Production of Self-Complementary Serotype 8 AAV Vector for Treatment of Hemophilia B

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 380 KB

english, 2010

849

669. Production of Lentiviral Vectors in Suspension Cells Using Recombinant Baculoviruses

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 380 KB

english, 2010

850

670. Increased Therapeutic Effect of Cell Transplantation for Wound Healing in Diabetic Mice by Prolonging the Survival of Transplanted Cells Using Adhesamine, a Synthetic Adhesion Molecule

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 441 KB

english, 2010

851

671. Characterization of Positioning Effect of pol III-shRNA Transcription Unit in scAAV Vector Genome on the Packaging Efficiency and Functionality of shRNA Silencing

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 232 KB

english, 2010

852

672. Rapid Production of High Titer Virus Using a Concentrated Retrovirus Expansion Device

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 252 KB

english, 2010

853

674. An Efficient and Complete Platform for the Production and Use of Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 232 KB

english, 2010

854

675. Development, Scale-Up and Technology Transfer for Rapid, Automated, cGMP Compliant, Large-Volume Suspension Cell Based Process for Lentiviral Vector Production

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 288 KB

english, 2010

855

676. Early Gestational Gene Transfer with Lentiviral Vector Driven by a Liver Specific Promoter Targets the Fetal Liver and Improves Phenotype in a Murine Model of Wilson's Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

856

677. A Combination of Pharmacological and Liver-Directed Gene Therapy Results in Long-Term Phenotypic Correction of a Mouse Model of Argininosuccinic Aciduria

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

857

678. Update of Stem Cell Gene Therapy Clinical Trial for ADA-Deficiency in Japan

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

858

680. AAV Vector Mediated Enzyme Replacement Therapy of Hypophosphatasia (HPP) Model Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 264 KB

english, 2010

859

681. rDNA-AAV Vectors for the Treatment of Phenylketonuria

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

860

682. Safety and Efficiency of Site-Specific Integration AAV-rDNA Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

861

683. Functional Improvement of LGMD Hamsters by AAV-hMG53-Mediated Sarcolemma Repair

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

862

685. The Principal Function of the Lentiviral Central DNA Flap Is Defense Against APOBEC3 Protein Restriction

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

863

687. “Same Day” Ex Vivo Regional Gene Therapy: A Novel Approach To Enhance Bone Repair

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 255 KB

english, 2010

864

688. The R Peptide Sequence of XMRV Envelope Glycoprotein Determined the Pseudotyping Specificity with HIV-1 and MLV Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 273 KB

english, 2010

865

689. Virus-Receptor Mediated Transduction of Dendritic Cells among Ross River Virus and Semliki Forest Virus Pseudotyped Lentivectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 255 KB

english, 2010

866

690. Effect of TLX1 Homeobox Gene Expression on Insertional Immortalization by an Insulated Self-Inactivating Retroviral Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 255 KB

english, 2010

867

691. Transcriptional Pulsing of Retroviral Transgenes in Mouse Embryonic Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

868

692. The Cellular Fibronectin Matrix Is a Key Factor for the Attachment of VSV-G Pseudotyped Vector Particles and Its Cell Type Specific Complexity Provides a Mechanism for Delayed Cell Entry

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

869

693. Targeting of Lentiviral Vectors and Positioning of Transduced Cells by Magnetic Nanoparticles and Magnetic Field

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

870

694. Pseudotyped Hepatitis C Virus Particle Could Be a Liver Target Bio-Missile

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

871

695. Regulation of Cytokine Expression by Lentivirus-Encoded RNA Antagomirs

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

872

696. Effects of Replicators on Retroviral Variegation and Extinction

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

873

697. Late Gestation Intrauterine Gene Transfer of AAV9 in Non-Human Primates Preferentially Transduces the Central Nervous System, Liver, Skeletal and Cardiac Musculature

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

874

698. Integrin-Linked Endocytosis Directs AAV2 and AAV1 to a Highly Efficient Transduction Pathway

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

875

699. Computationally-Guided Rational Design of Tropism-Modified AAV Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 322 KB

english, 2010

876

700. Identification of Human Hepatocyte Growth Factor Receptor as a Cellular Receptor/Co-Receptor for AAV3

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 367 KB

english, 2010

877

701. Rapid Transgene Expression and Long-Term Phenotypic Correction of a Lethal Mouse Model of Methylmalonic Acidemia Using rAAV9-Mediated Gene Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

878

702. Widespread Transduction of Brain Parenchyma after Intraventricular Injection of AAV9

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

879

703. AAV1.9-3 Exhibiting Significantly Impaired Infectivity and Substantially Delayed Blood Clearance, Is a Novel Detargeted Platform of AAV Random Peptide Display Libraries

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 353 KB

english, 2010

880

704. Development of the D[+]-Sequence-Deleted ssAAV2 Vectors: Enhanced Transgene Expression In Vitro and In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 335 KB

english, 2010

881

705. Influence of Murine Parvovirus on Adeno-Associated Virus Vector-Related Expression in Murine Models

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 446 KB

english, 2010

882

706. Involvement of the Glucocorticoid Receptor Signaling in AAV2 Vector-Mediated Transgene Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 313 KB

english, 2010

883

707. AAV9-Mediated Catalase Expression in Mitochondria Improves Exercise Performance in mdx Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 313 KB

english, 2010

884

708. Development of a Liver Specific Tet-On Inducible System for AAV Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 331 KB

english, 2010

885

709. Intracortical Delivery of an Adeno-Associated Viral Vector Expressing the Glutamate Transporter (GLT1) Decreases Stroke-Induced Extracellular Glutamate Overflow and Cerebral Infarction after Transient Middle Cerebral Artery Occlusion in Rats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

886

710. The Potential Role of Chromatin Structure in Adeno-Associated Virus Type 2 Locus-Specific Integration at AAVS1

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

887

711. Augmentation of Traditional Ad/AAV Helper Plasmids with HPV E1 Increases rAAV DNA Replication and Virus Production in 293 Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 485 KB

english, 2010

888

712. Hexon-Modifications of hAd5 Based Helper-Virus for High-Capacity Adenoviral Vector Production and Limitations Imposed by HVR-Exchanges Utilizing Serotype Switch

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 460 KB

english, 2010

889

713. Use of Cell-Penetrating Peptides To Induce a Bystander Effect

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 459 KB

english, 2010

890

714. Highly Sensitive Detection of Prostate Cancer Cells In Vivo by a Dual Adenoviral TSTA Trans-Amplification System

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

891

715. Coagulation Factor X-Mediated Adenoviral Transduction Is Dependent on Heparan Sulfate Proteoglycan Sulfation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

892

716. Towards the Generation of a Baculovirus Display Library for Cell Targeting

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 254 KB

english, 2010

893

717. Broad Spectrum Activity of the HSV-2 Vector ΔRR Enhances Neuroprotection and Includes Crosstalk between Neurons and Glial Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

894

720. Adenovirus-Mediated Decorin Expression Induces Cell Death through Activation of p53 and Mitochondrial Apoptosis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

895

721. PEG-Modified Adenovirus Conjugated with Herceptin Improves Therapeutic Value by Active Targeting through Her2/neu with an Enhanced Safety Profile

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

896

722. Study on Testing Method for Adeno-Associated Virus (AAV) of Adventitious Virus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

897

723. Ad5-Ad48 Hexon Hypervariable Region Substitutions Affect Viral Biodistribution and Inflammatory Responses In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 333 KB

english, 2010

898

724. Evaluation of a Porcine Adenovirus 3 Vector as a Post-Exposure Vaccine Candidate

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 333 KB

english, 2010

899

725. The Knob Domain Replacement Strategy Is Broadly Applicable to a Wide Variety of Adenovirus Fiber Proteins

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 352 KB

english, 2010

900

727. Duration of Expression from CpG-Free Plasmids Following Hydrodynamic Delivery to the Mouse

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

901

728. A Liver-Specific Promoter Enhances Expression of alpha-L-Iduronidase in Sleeping Beauty Transposon-Mediated Gene Therapy of Murine MPS I

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

902

729. New Generation, S/MAR Based, Episomal Vectors with High Potential for Gene Transfer into Hematopoietic Progenitor Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

903

730. High-Yield, Non-Viral Production of Engineered Cells through Sleeping Beauty Transposon-Mediated Gene Integration Coupled with Selective Amplification

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 249 KB

english, 2010

904

732. Using Helper Virus-Free Packaged HSV-1/ EBV Hybrid Amplicons To Induce Directed Cell Motility toward SDF-1α

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

905

733. A Novel System for the Evaluation of Genotoxicity for Gene Therapy Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 251 KB

english, 2010

906

735. Cells Labeled with Fatty Acid-Oligonucleotide Conjugates as Potentially Suitable for Tissue Engineering

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 315 KB

english, 2010

907

736. An Enhanced Non-Viral Expression Vector (EEV) - For Cancer Treatment

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 299 KB

english, 2010

908

738. Improved Antibiotic-Free Plasmid Vector Design by Incorporation of Transient Expression Enhancers

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 423 KB

english, 2010

909

739. Genomic DNA Reduction for Therapeutic DNA Manufacture

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 296 KB

english, 2010

910

740. Preclinical Efficacy and Safety of Electroporation-Mediated Delivery of a Plasmid Vector Manufactured in the Absence of Antibiotic Selection

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 296 KB

english, 2010

911

741. Hepatic Stellate Cell-Specific TGF-β1 Gene Silencing for Treating Liver Fibrosis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 413 KB

english, 2010

912

742. Nuclear Enclosure of Nanospheres in Artificial Nuclei and in Living Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 338 KB

english, 2010

913

743. Analysis of Intracellular Trafficking of MPC-Coated Multifunctional Envelope-Type Nano Device (MPC-MEND) in Isolated Hepatocyte

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 372 KB

english, 2010

914

744. Photo-Activated Release of DNA from Liposome Complexes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

915

746. Modifying DNA Nanoparticles with Biotin for Versatile Ligand Substitution

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

916

747. An Integrative Approach To Monitor Gene Delivery to Airway Epithelium by Nanocomplex Vectors and Radioimaging Agents

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

917

748. Development of Small, Homogenous Non-Viral Gene Vector Using Mono-Cationic Detergents

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

918

749. Development of DNA Complex-Releasing Systems Using Biodegradable Apatite Cement and Therapeutic Effect in Tumor Model Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 426 KB

english, 2010

919

750. Investigation of Cross-Linking Peptides as Gene Delivery Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 444 KB

english, 2010

920

751. Rapid and Highly Efficient Magnetofection of IL-10 to Extremely Susceptible Vascular Endothelial Cells Using Hybrid Iron Oxide-PEI Conjugate

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 203 KB

english, 2010

921

752. Cocaine Conjugated to Disrupted Adenovirus - A Synthetic/Viral Protein Hybrid Strategy To Elicit High Titers Against Non-Immunogenic Addictive Small Molecules

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 203 KB

english, 2010

922

754. Optimization of Sendai F/HN Viroplexes for Efficient Transfection of Plasmid DNA and siRNA to Suspension Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

923

755. Comparative Evaluation of Disulfide-Linked and Hydrophobically-Modified PEI for Plasmid Delivery In Vitro

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 364 KB

english, 2010

924

756. Linear Polyethylenimine Prepared by Partial Acid Hydrolysis of Poly (2-ethyl-2-oxazoline) for DNA and siRNA Delivery In Vitro

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 347 KB

english, 2010

925

757. Impact of Counterion and Hydrophobic Anchor on Physicochemical Characteristics and Transfection Efficiency for a Series of Pyridinium Cationic Lipid-Based Lipoplexes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 347 KB

english, 2010

926

758. AAV-Mediated Liver Gene Transfer Results in Long-Term Expression of Therapeutic Levels of ARSB in MPS VI Cats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 347 KB

english, 2010

927

759. Beta-Glucuronidase Expressed from AAV Is Poorly Modified with Mannose-6-Phosphate Residues and Fails to Completely Correct Mucopolysaccharidosis VII in Dogs

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

928

760. Gene Transfer To Induce Cellular Reprogramming for the Treatment of Diabetes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

929

761. Salivary Gland hPTH Gene Transfer: Interesting Possibilities

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

930

762. Efficient Gene Transfer to the Pancreas Using ssAAV for Prevention of Type 1 Diabetes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

931

763. Tolerance Induction to Coagulation Factor IX Using Gene Modified Primary B Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

932

764. Microporous Polymer Scaffolds for Islet Transplantation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

933

765. AAV2- and AAV9 -Mediated Gene Transfer of LDL Receptor to Hypercholesterolemic WHHL Rabbits

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

934

766. Hairpin Ribozymes Encoded in Adenoviral Vectors Reduce Drinking in Alcohol Dependent Rats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 226 KB

english, 2010

935

767. Sustained Therapeutic Levels of FVIII Activity from Lentivirally-Engineered Blood Outgrowth Endothelial Cells Requires Cell Stability and Augmented Angiogenesis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

936

770. Layered Scaffolds for Islet Transplantation and Gene Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

937

771. Lentiviral Vector Correction of Fabry Disease Encompassing a Cell Fate Control Safety Element

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

938

772. Wolman Disease (LIPA p.G87V) Genotype Frequency in Patients of Iranian-Jewish Descent

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

939

773. Delayed Inhibition of Soluble Tumor Necrosis Factor Signaling Blocks the Progressive Phase of Dopaminergic Neuron Death in a Rat Model of Parkinson's Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 210 KB

english, 2010

940

774. Identification and Analysis of a Novel Y-Secretase Inhibitory Gene

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 265 KB

english, 2010

941

775. Effects of Alpha 7 Nicotinic Receptor Gene Delivery on Processes Underlying Cell Viability and Cell Differentiation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

942

776. Delivery of hGDNF to Brain Using DNA Nanoparticles: Neuroprotection of the Nigrostriatal Pathway

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

943

777. ProSavin, a Gene Therapy for Parkinson's Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

944

779. Delivery Co-Interventions in Gene Transfer and Cell Transplantation: A Meta-Analysis of Surgical Risk for Trials Involving Inoculation to Deep Brain Structures

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

945

781. HSV Gene Transfer of a Poreless TRPV1 Receptor Reduces Pain-Related Behavior in Rodents

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

946

782. A Comparison of AAV Serotypes and Routes of Administration for Delivery to the Spinal Cord

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

947

783. Engineered Zinc Finger Protein Improves Microvascular Recovery, Neuronal Sparing and Functional Recovery in Modeled Traumatic Brain Injury in Rats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

948

784. Blocking the Myostatin Signal Improves the Success of Human Myoblast Transplantation in Dystrophic Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

949

786. Comparison Studies of rAAV6 Transduction Efficiency in Adult and Aged Muscles

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

950

787. Gene Transfer of Signals Critical for Allograft Healing Using Immobilized Freeze Dried Adeno-Associated Viral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 285 KB

english, 2010

951

788. Rescue from Respiratory Dysfunction by Transduction of Full-Length Dystrophin to Diaphragm Via the Peritoneal Cavity in Muscular Dystrophy Mouse

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

952

789. Restoration of Wild-Type DMD mRNA in an Immortalized Human Fibroblast Line from an Exon 8-9 Duplicated DMD Patient

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

953

790. Expression of Micro-Dystrophin and Full-Length Dystrophin in Mouse and Dog Muscles

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 278 KB

english, 2010

954

793. Gene Transfer-Driven Inhibition of Cardiac Ankyrin Repeat Protein as a Potential Therapy for the Correction of Muscular Dystrophies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 287 KB

english, 2010

955

794. Background Strain and Natural Selection Improves Survival of HIBM Mouse Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 210 KB

english, 2010

956

795. Combining Stem Cells and Exon Skipping Approach To Treat Muscular Dystrophies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 210 KB

english, 2010

957

796. Gene and Cell Therapy Approaches Tto Treating the Dysferlinopathies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 210 KB

english, 2010

958

797. Delineation of RNA Antisense env Effects on HIV Quasispecies Recovered from Lexgenleucel-T Treated Subjects: Influences of Sequence Similarity of the RNA Antisense with the Targeted HIV env Transcript and VRX496-Modified Cell Persistence

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

959

798. Assessing Preclinical Safety of a CCR5-Specific Zinc Finger Nuclease in Human CD4+T Cells Using the NSG Immunodeficient Mouse Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 210 KB

english, 2010

960

799. Multivirus-Specific T Cell Immunotherapy To Prevent or Treat Infections of Allogeneic Stem Cell Transplant Recipients

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 210 KB

english, 2010

961

800. Inhibition of HIV-1 Replication in VRX496-Modified CD4 T Cells: Importance of Cytoplasmic dsRNA Components

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

962

801. Engineering Resistance to CXCR4-Tropic HIV: A Cutting Edge Approach

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

963

802. Development of an Unbiased Method for Characterizing Viral Populations Using Next Generation Sequencing Technology

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

964

803. Long Term Persistence of VRX496-Modified CD4+ T Lymphocytes Following Lexgenleucel-T Infusions for the Treatment of HIV/AIDS: Results from a Phase 2 Clinical Trial

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

965

804. Generation of Restriction Factor-Transgenic Cats Using Lentiviral Transgenesis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

966

805. Combinatorial Latency Reactivation for HIV-1 Subtypes and Variants

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 232 KB

english, 2010

967

806. Highly Efficient Expression and Purification of Hepatitis C Virus Core Protein in E.coli

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 203 KB

english, 2010

968

807. Molecular Epidemiology of HCV Infection among Recovery and Chronic Blood Donors in Guangdong, China

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 203 KB

english, 2010

969

808. Occult Hepatitis B Virus Infection in Shenzhen, China

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 203 KB

english, 2010

970

810. Oxaliplatin Increases the Antitumor Effect of Vector-Delivered Interleukin-12 Expression in a Murine Model of Hepatic Colorectal Cancer Metastases

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 323 KB

english, 2010

971

811. Optimizing Dendritic Cell Vaccination by Combination with Oncolytic Adenovirus Coexpressing Interleukin-12 and Granulocyte-Macrophage Colony Stimulating Factor

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 382 KB

english, 2010

972

812. Bacterial Vector Mediated Vaccination for Prostate Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

973

813. Evaluation of Long-Term Transgene Expression in piggyBac -Modified Human T Lymphocytes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

974

814. Engineered Drug Resistant Immunocompetent Cell Therapy for Neuroblastoma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 257 KB

english, 2010

975

816. Tumor Antigen Delivery to Secondary Lymphoid Organs by Interleukin-12 Gene Modified T Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 315 KB

english, 2010

976

817. Pluronic Based Formulations Are Promising Anticancer DNA Vaccines

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 279 KB

english, 2010

977

818. Efficient Induction of Humoral and Cellular Immune Responses Against Her-2/neu by Combination of Naked DNA and Adenoviral Vectors Expressing Genetically Engineered Her-2/ neu

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 279 KB

english, 2010

978

820. Potent Transfer of Human CD40L to Chronic Myeloid Leukemia Stem Cells Via an AdCD40L Genetically Engineered Bystander Cell Line

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 501 KB

english, 2010

979

821. Abstract Withdrawn

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 501 KB

english, 2010

980

822. Preclinical Efficacy Data of MGN1601, a Tumor Vaccine Comprising 4-Fold Gene-Modified and Irradiated Allogeneic Tumor Cells in Combination with a DNA-Based Immunomodulator for the Treatment of Metastatic Renal Carcinoma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 528 KB

english, 2010

981

823. Electrically Mediated Delivery of Plasmid DNA-Encoding Cytokines for the Generation of an Enhanced Immune Response Against Melanoma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 250 KB

english, 2010

982

824. Cytokine-Based Log-Scale Expansion of Functional Human Dendritic Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 249 KB

english, 2010

983

826. Imiquimod, a TLR7 Agonist, Delays Brain Tumor Growth through Suppression of Treg in Brain

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 249 KB

english, 2010

984

827. Strategy Escalation: An Emerging Paradigm for Safe Clinical Development of T Cell Gene Therapies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 250 KB

english, 2010

985

828. Combination of Promoter and microRNA Detargeting Is Greatly Advantageous in Attenuation of Adenoviral Replication in Hepatic Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 288 KB

english, 2010

986

829. Combining Oncolytic Herpes Simplex Virus and Chemotherapy: G47Δ and Temozolomide Synergize in Killing Glioma Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

987

830. Transcriptional Targeting of an Oncolytic HSV-1 to Midkine-Positive Tumors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

988

831. Preclinical Studies Support the Efficacy of Prodrug Activator Gene Transfer in Experimental Glioma Models by Enhanced Replication-Competent Retrovirus Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

989

832. Intrapleural Administration of Third Generation Oncolytic HSV-1 (G47Δ) Is Highly Efficacious in Orthotopic Malignant Pleural Mesothelioma Models

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

990

833. Engineered Measles Viruses for Oncolytic Therapy of Pancreatic Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

991

834. Incorporation of a Mutated E1A and a Chimeric 5/3 Fiber Improved the Therapeutic Efficacy of a Novel CRAd (Ad-F512) on Disseminated Ovarian Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

992

835. Inhibition of Autophagy Has Negative Impact on Virus Production

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

993

837. Determining Adenovirus Titers Following Extraction of the Virus from Tumors in the Presence of Anti-Adenovirus Antibodies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

994

838. Abstract Withdrawn

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

995

839. The Use of Third Generation Oncolytic HSV-1 Expressing Luciferase for Demonstration of Real-Time Biodistribution and Dynamics

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 265 KB

english, 2010

996

840. Highly Attenuated Vaccinia Virus as a Potential Oncolytic Agent for Cancer Virotherapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

997

842. Increased Oncolytic Ability of a Double RGD-Modified Adenovirus in Ovarian Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

998

843. Increasing the Efficacy of Monoclonal Antibody Therapy of Breast Cancer through Transient Remodeling of Intercellular Junctions

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

999

844. Improvement of Cancer Gene Therapy by Incorporating a Chicken Hypersensitive Site-4 Insulator into TSTA-Based Tumor-Targeting Vector

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

1000

845. Cytokine Induced Killer (CIK) Cells for Cell Therapy of Acute Myeloid Leukemia (AML): Improvement of Their Immune Activity by Expression of CD33-Specific Chimeric Receptors (CR)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

1001

846. Exosome-Mediated Suicide Gene Therapy for Brain Tumors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

1002

847. Eradication of Therapy-Resistant Human Prostate Tumors Using an Ultrasound Guided Site-Specific Cancer Terminator Virus Delivery Approach

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 347 KB

english, 2010

1003

848. Systemic Tumour Targeting with Orally Administered Non-Pathogenic Bacteria

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 328 KB

english, 2010

1004

849. Transient Intratumoral Relaxin Expression Results in Tumor Stroma Degradation and Improves Breast Cancer Therapy by Herceptin

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 353 KB

english, 2010

1005

850. Developing a Multi-Modal Gene-Based Imaging and Therapy To Treat Disseminated Hormone Refractory Prostate Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

1006

851. Adenoviruses-Mediated Expression and Cell-Mediated Delivery of Interferon-Lambda Produced Anti-Tumor Effects to Human Esophageal Carcinoma In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

1007

852. Inhibition of Angiogenesis and Suppression of Colorectal Cancer Metastatic to the Liver Using the Sleeping Beauty Transposon System

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

1008

853. Plasmid AMEP, a New Efficient Antitumor Gene Transfer Product for the Treatment of Metastatic Melanomas

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

1009

854. Adenovirus-Delivered Short Hairpin-Structured RNA for Microphage Migration Inhibitory Factor Silencing Inhibits Human Prostate Cancer Cell Proliferation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 383 KB

english, 2010

1010

855. Monitoring of Electroporation-Mediated Diphtheria Toxin A Expression in Prostate Cancer Cells Grown in SCID Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 383 KB

english, 2010

1011

856. Clinical Gene Transfer in the Community: Ten Years of Local IBC Review

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 465 KB

english, 2010

1012

858. Successful Treatment of Canine Leukocyte Adhesion Deficiency Using a MSCV-Promoter Based Foamy Viral Vector and Low-Dose Busulfan Conditioning

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 338 KB

english, 2010

1013

859. Genetically Engineered VIP-Expressing Dendritic Cells Are Therapeutic in the Autoimmune Mouse Model of Experimental Autoimmune Encephalomyelitis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

1014

860. Long-Term Preclinical Evaluation of Lentiviral Vectors for SCID-X1 Gene Therapy in a SCID-X1 Mouse Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

1015

862. Prevention of Atopic Dermatitis by Plasmid-Based Sustained Transgene Expression of Interferon γ in NC/NGA Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

1016

863. Expression of an Antibody Against CTLA4 from Tumor Infiltrating Leukocytes Induces Efficient Anti-Tumor Immune Responses in a Mouse Model for Breast Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 255 KB

english, 2010

1017

864. Foamy Virus Vectors with MGMT-P140K Mediate Efficient Selection in a Canine Pyruvate Kinase Deficiency Model for Hematopoietic Stem Cell Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1018

865. Modeling of Congenital Erythropoietic Porphyria by RNA-Interference: A New Tool for Preclinical Gene Therapy Evaluation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1019

866. Gene Corrected Neutrophils from Induced Pluripotent Stem Cells Derived from an X-CGD Disease Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

1020

867. The Comparison of Various Promoters for Wiskott Aldrich Syndrome Gene Therapy Using Insulated Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1021

868. Sleeping Beauty -Mediated Therapeutic Gene Transfer into Hematopoietic Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1022

869. Myelospecific Self-Inactivating Gammaretroviral Vectors for p47phox CGD Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1023

871. Modulation of CXCR4 Expression by Novel Growth Factor Cocktail on Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1024

873. SIV Vector Pseudotyped with SeV-F/ HN Envelope Proteins Produces Long Lasting Expression in the Murine Lung, Is Readministrable and Transfects Human Airway Models

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1025

874. Airway Lentiviral Gene Transfer In Marmosets

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1026

875. Pulmonary Delivery of mRNA: In Vitro and In Vivo Evaluation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1027

876. Inhibiting Bap31 by Dominant Negative p20 Does Not Promote CFTR Translocation to the Cell Membrane

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1028

877. Targeted siRNA Delivery to Lung Epithelia Reduces Mucus Secretion in a Mouse Model for Allergic Asthma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1029

878. The T1α Promoter Mediates Nuclear Import of Plasmid DNA into Alveolar Epithelial Type I Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1030

879. Effects of LPC on Lung Mechanics in Non-Human Primates

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1031

881. Pseudotype Switching as a Strategy To Permit Efficient Re-Administration of Lentiviral Vectors to Mouse Airways

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1032

882. Antinociceptive Potentiation and Attenuation of Tolerance by Intrathecal β-Arrestin 2 siRNA in Rats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1033

883. siRNA Cationic Delivery Systems for Therapeutic Applications

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

1034

884. Therapeutic Effect of Intravenous Administration of New Type Hybrid Staple-Modified NFkB Decoy Oligonucleotides on Inflammatory Bowel Disease Model Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1035

885. Artificial miRNA Clusters for Targeting Multiple Regions of the HCV Genome

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1036

888. Parallel Assessment of Silencing Potency of Conventional SiRNAs vs. SiRNA Variants

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

1037

889. Inhibition of Viral Hepatitis with Non-Coding RNAs

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 266 KB

english, 2010

1038

891. A Combination of PHD2 Prolyl Hydroxylase Silencing and Biocompatible Polyplex Nanocarrier Enhances Therapeutic Angiogenesis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

1039

892. Development of a Novel, RNAi-Based Therapeutic Targeting Pancreatic Duodenal Homebox-1 (PDX-1) for Insulinoma and Pancreatic Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1040

893. Systemic Delivery of Highly Active Functionalized Lipopolyamine-Formulated siRNA to Murine Lung with Reduction of Inflammation by Dexamethasone Pre-Treatment

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1041

894. Ornithine and Tryptophan as an Efficient Polycation for siRNA Delivery to Tumor Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

1042

895. Influence of 5’ Terminal Triphosphate in Short Hairpin RNA Targeting HCV Replicon on Antiviral Activity and Innate Immune Response

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1043

896. Enhanced Delivery of GFP siRNA Using New Amino Lipid-Based Cationic Liposomes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1044

898. Engraftment into the Liver of Transplanted Adipose Tissue-Derived Stromal Cells Detected by Noninvasive Bioluminescence Imaging

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1045

899. Establishment of iPS Cells Derived from the New World Monkey of Common Marmoset

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1046

900. Umbilical Cord Blood-Derived Mesenchymal Stem Cells Acquire Cardiac Phenotype with Oxytocin Treatment and Enhance Cardiac Recovery after Myocardial Infarction

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1047

901. Ex Vivo Expansion and Enrichment of Murine Adult Neural Stem Cells for Gene Therapy of Neurogenetic Diseases

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1048

902. Dual Effect of Mesenchymal Stem Cells Genetically Modified with a Single-Chain Antibody Against EGFRvIII on the Targeting and Growth of EGFRvIII Expressing Experimental Glial Tumors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

1049

903. Expression of Coagulation Factors from Murine iPS-Derived Liver Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

1050

904. The Granules Isolated from Mast Cells Have Increased the Survival of Bone Marrow-Derived Mesenchymal Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

1051

905. Large-Scale Expansion of Undifferentiated Human Embryonic Stem Cells in Serum-Free Suspension Culture

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 266 KB

english, 2010

1052

906. Enrichment of Bone Marrow Stem Cells In Vivo by Dissociation- and Proliferation-Inducing Compounds Augments Bone Growth in a Mouse Segmental Bone Defect Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

1053

907. A Delayed Treatment of Mesenchymal Stem Cells Is Effective in Rescuing Mice with Established LPS-Induced Acute Lung Injury

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

1054

908. Lentiviral Vector-Mediated Transduction in Murine SMA Embryonic Stem Cells Derived from Spinal Muscular Atrophy (SMAI) Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 293 KB

english, 2010

1055

909. Development of a Reporter System for I-SceI-Mediated Genome Editing in Canine Hematopoietic Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 217 KB

english, 2010

1056

910. Liver Disease Correction with Cells Isolated from Cadaveric Liver

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 217 KB

english, 2010

1057

911. Efficient Gene Transfer into CD34+ Hematopoietic Stem Cells Using Clinical Grade Lentiviral Vector Expressing the Human Beta-Globin Gene

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

1058

912. Embryonic Stem Cell Therapy Enhances Tendon Regeneration in a Large Animal Flexor Tendonitis Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 217 KB

english, 2010

1059

913. Generation of Cystic Fibrosis Induced Pluripotent Stem (iPS) Cells for Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 217 KB

english, 2010

1060

4. AAV Capsid Sequence Significantly Influences Intracellular Viral Trafficking in Polarized Human Airway Epithelial Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 257 KB

english, 2010

1061

6. Novel Cardiac-Tropic AAV Vectors by DNA Shuffling and In Vivo Screening

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 257 KB

english, 2010

1062

10. Targeted Transgene Integration in Epidermal Stem Cells by Zinc Finger Nucleases and Integrase-Defective Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 317 KB

english, 2010

1063

18. Adeno-Associated Virus Vector Expressing an Amyloid beta 1–42 Intrabody Rescues White Matter Pathology during Early Alzheimer's Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

1064

22. Combination Therapy for the Treatment of Infantile Neuronal Ceroid Lipofuscinosis (INCL)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 275 KB

english, 2010

1065

42. Intensive Pharmacological Immunosuppression Allows for Repetitive Liver Gene Transfer with Recombinant Adenovirus in Nonhuman Primates

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

1066

45. Impact of Pre-Existing Neutralizing Antibodies on AAV8-Mediated, Liver-Directed Gene Transfer and Vector Biodistribution in Nonhuman Primates

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

1067

50. Triplex-Induced Correction of IDUA Gene Mutations as a Novel Hurler Syndrome Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

1068

58. Chimpanzee-Derived Novel Natural Variants of AAV9: Vector Development and Interrogation of Correlations between Capsid Structure and Vector Biology

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

1069

60. Enhancing AAV2 Transduction by Anti-Microtubule Drugs through RhoA GTPase Activation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 266 KB

english, 2010

1070

63. Effects of DNA Methylation on AAV Integration and Transgene Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 274 KB

english, 2010

1071

75. Intrathecal Injection of HDAd Vectors Results in Long-Term Transgene Expression in Neuroepithelial Cells and Neurons

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

1072

81. FX Domain Fused to Single-Chain Antibodies Improve Oncolytic Effect of Ad5 In Vitro and In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 205 KB

english, 2010

1073

91. Cell Synchronization by Serum Shock Facilitates rAAV-Mediated Gene Targeting

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 251 KB

english, 2010

1074

92. Critical Amino Acid Residues within the PhiC31 Integrase DNA Binding Domain Affect Recombination Activities in Mammalian Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

1075

93. Chromatin Structure of Two Genomic Sites for Targeted Transgene Integration in Induced Pluripotent Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

1076

108. Hydrodynamic Gene Delivery Efficiency Is Reduced in Fibrotic Liver in Rats

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 243 KB

english, 2010

1077

112. A Study on Techniques for Safety Assessment of Gene Therapy Products Using Electroporation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

1078

117. Modular Ternary Polyacridine DNA Polyplexes for Targeted Gene Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 287 KB

english, 2010

1079

130. Electrospun Fibers with the Core-Loading of DNA and Poly(ethyleneimine) Complex as Potential Tissue Engineering Scaffold

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 346 KB

english, 2010

1080

136. Efficacious Gene Therapy in Pompe Disease Requires Mannose-6-Phosphate Receptor Expression

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

1081

153. Long Term Antisense Treatment in Dystrophic Mouse Models for Duchenne Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

1082

157. Manganese Superoxide Dismutase Plasmid/Liposome Complex (MnSOD-PL) and Mitochondrial Targeted Nitroxide JP4–039 Ameliorate Irradiation-Induced Delay in Bone Wound Healing

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

1083

178. IL-15 Reduces Suppressive Effects of Regulatory T Cells (Tregs) on Antigen-Specific Cytotoxic T Lymphocytes (CTLs)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1084

181. Key Role of B Cell as Antigen Presenting Cells in the Anti-Tumor Immunity Induced by a Combined Immune-Stimulatory/Conditional Cytotoxic Therapy for Glioblastoma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1085

183. Adoptive Transfer of T Cells Genetically Targeted to the MUC16 Antigen Eradicates Established Peritoneal Ovarian Tumors in SCID-Beige Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 246 KB

english, 2010

1086

186. Inhibition of Mice Colon Tumor Growth by Il-15 Immunogene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 245 KB

english, 2010

1087

188. Combined Gene Therapy Using Adenovirus Expressing Interleukin-12, Granulocyte-Macrophage Colony-Stimulating Factor, and Thymidine Kinase with Prodrug Ganciclovir

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

1088

189. Safety Data of MGN1601, a Tumor Vaccine, Made of Allogeneic, Transfected and Irradiated Tumor Cells in Combination with an Immunomodulator for the Treatment of Metastatic Renal Cell Carcinoma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

1089

195. Mechanisms of Cyclophophamide Enhancing Anti-Tumor Effects of Oncolytic Vesicular Stomatitis Virus for Breast Cancer Treatment

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 214 KB

english, 2010

1090

196. Clinical Characterization of the Adenovirus Death Protein-Potentiated Oncolytic Adenovirus VRX007

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 196 KB

english, 2010

1091

220. Modeling X-Linked Chronic Granulomatous Disease Using Neutrophils Differentiated from Patient-Derived Induced Pluripotent Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

1092

226. Development of B-Lineage Specific Lentiviral Vectors for Use in Gene Therapy for B-Cell Disorders

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 268 KB

english, 2010

1093

232. Ex Vivo Lentiviral Transduction of the Human Purine Nucleoside Phosphorylase (PNP) Gene in Bone Marrow Derived Hematopoietic Stem Cells: An In Vivo Model of Gene Therapy for PNP Deficient T-Cell Immunodeficiency

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1094

235. A Dual Self-Inactivating Lentiviral Vector System for Mifepristone-Inducible Bicistronic Transgene Expression in Primary Neural Progenitor Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 256 KB

english, 2010

1095

247. Systemic Administration of Bifunctional shRNA Lipoplex Targeting PDX-1 for Murine Model of Insulinoma and Pancreatic Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1096

265. Ovarian Cancer Stem Cells Are in an Epithelial/Mesenchymal Hybrid State and Have Multipotential Capacity for Differentiation

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

1097

271. A Novel Fluorescence/Fluorine MRI Dual-Mode Reagent for Cell Tracking and Determination of Cell Fate

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 421 KB

english, 2010

1098

274. Process Improvements for cGMP Manufacture of Lexgenleucel-T for HIV Treatment

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 248 KB

english, 2010

1099

283. Therapeutic Transgene Expression in Patient-Specific iPS Cells after Selection of “Safe Harbors” for Lentiviral Vector Integration

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 284 KB

english, 2010

1100

289. Therapeutic DNA Vaccination Followed by Standard-of-Care Therapy in Patients with Chronic Hepatitis C: A Rapid Clearance of Viremia

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1101

308. High-Throughput Sequencing of AAV Vector Integrants To Assess Potential for Genotoxicity

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

1102

315. Therapeutic Efficacy of Hematopoietic Stem Cell Gene Therapy for Hurler Type 1 Mucopolysaccharidosis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

1103

341. Engineered Human Regulatory T Cells Expressing Lentiviral PDL1 under Cell Fate Control Prevent Lethal Xenogeneic GVHD

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

1104

343. Lentiviral Vectors Containing an Endogenous Btk Promoter Restore B and Myeloid Lineage Expression and Function in a Murine Model of X-Linked Agammaglobulinemia

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 210 KB

english, 2010

1105

347. Genome Wide Characterization of Insertion Profiles of Integration Deficient Lentiviral Vectors Using Two-Sided Non-Restrictive and Standard LAM-PCR

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 218 KB

english, 2010

1106

351. Clonal Analysis Allows Characterization and Risk Assessment of Viral-Cellular Fusion Transcripts Generated by Transcriptional Termination Read-Through in Keratinocytes Transduced with Self-Inactivating Retroviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

1107

354. Development of a pCL20c-Backbone Based HIV Lentivirus Vector That Achieves Efficient Functional Correction of Neutrophils Arising from Transduced CD34+ Hematopoietic Progenitor Cells of Patients with X-Linked Chronic Granulomatous Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 263 KB

english, 2010

1108

358. Lentiviral Microarrays for High-Throughput and Real-Time Monitoring of Gene Expression Dynamics

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 382 KB

english, 2010

1109

364. Multifunctional Peptide Ligands for AAV Capsid Recognition

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 335 KB

english, 2010

1110

370. Strategies To Achieve Enhanced Transduction of Primary Human Dendritic Cells by AAV2 Vectors and Reduced Cytotoxic T Cell Response to Viral Capsids and Transgene Products

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 598 KB

english, 2010

1111

373. Improved and Persistent Expression with Novel AAV Serotypes for Neonatal Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 384 KB

english, 2010

1112

374. Optimization of AAV Vectors for Improved Therapeutic Protein Expression in the Canine Models for Duchenne Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 462 KB

english, 2010

1113

383. Capacitance-Based Real Time Monitoring of Virus Transduction

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 213 KB

english, 2010

1114

389. Dual-Expression Adenoviral Vectors as Vaccine Carriers

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

1115

391. Human Adenovirus Type 40 Vector Engineering for Intestinal Mucosa Targeting

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

1116

395. In Vitro and In Vivo Transfection Efficiency of DNA Nanoparticles Made with Histidinylated lPEI and Plasmid DNA Containing NFκB DNA Binding Sequence

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1117

396. Intracellular Trafficking and Decondensation Kinetics of Chitosan-pDNA Polyplexes

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1118

398. Tumour Therapy with Listeria Monocytogenes Vector-Mediated DNA Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

1119

399. Efficient Gene Transfer to Umbilical Cord Blood-Derived Mesenchymal Stem Cells Mediated by MR Imageable Inorganic-Organic Hybrid Conjugate

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

1120

401. MHC Class-I Mediated Antigen Presentation by a Non-Viral T-MEND Vector Expressing a High Luciferase Activity in Murine Derived Dendritic Cells (JAWS-II)

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 236 KB

english, 2010

1121

408. Lentiviral Vector-Transduced Hematopoietic Stem Cell Gene Therapy for Cystinosis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 489 KB

english, 2010

1122

412. AAV8 Mediated Expression of LDLR Ameliorates Hypercholesterolemia and Regresses Pre-Existing Atherosclerosis in a Humanized Mouse Model of Familial Hypercholesterolemia

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1123

414. Gene Therapy Ex Vivo in the ADA-Deficient Mouse: Increased Survival and Efficacy with Short-Term Enzyme Replacement Therapy Post Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 244 KB

english, 2010

1124

422. Development of HIV-1 Based Lentiviral Vectors Expressing Pre-Trans-Splicing Molecules (PTMs) for Liver-Directed Therapies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 369 KB

english, 2010

1125

427. Strategies for Promoting Survival of Transplanted Mesenchymal Stem Cells in a Myocardial Infarction Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 645 KB

english, 2010

1126

452. Efficacy of an Adenovirus Vector/Cocaine Conjugate-Based Anti-Cocaine Vaccine in Shielding the Brain from Systemically Administered Cocaine

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

1127

457. Increased Muscle Regeneration in Hindlimb and Diaphragm of mdx Mice Treated with AAV9 Mini-Dystrophin and Octalysine-NEMO Binding Domain Peptide

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 281 KB

english, 2010

1128

472. Combining mTor Inhibitors with Rapamycin-Resistant T Cells: A Two-Pronged Approach to Tumor Elimination

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1129

475. Emozolomide Inhibits the Migration of Immune Cells into the Brain Tumor Microenvironment and Their Systemic Expansion, but Exerts a Synergistic Anti-Tumor Effect When Combined with Combined Conditional Cytotoxic/Immune-Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 241 KB

english, 2010

1130

481. Combination Therapy for Melanoma Using Virotherapy and Alternate TLR Stimulating Agonists

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1131

486. AdCD40L Gene Therapy Enhances the Efficacy of Chemotherapy in Experimental Bladder Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 260 KB

english, 2010

1132

499. Activity of Oncolytic Measles Virus Strains Against Brain Tumor Stem Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

1133

501. Oncolytic Adenovirus with Somatostatin Motif for Selective Infection of Neuroendocrine Tumors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

1134

503. Safety of Intravenous Ad5-Mda-7, a Potential Anti-Metastatic Agent in Gynecologic Cancer

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

1135

508. Mechanisms of the Tmpk/AZT-System Bystander Effect; Cell Fate Control Gene Therapy for Anti-Cancer Treatment

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 196 KB

english, 2010

1136

520. Regulatory Submission Process for Gene and Cellular Therapies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

1137

524. Successful Treatment of Canine Leukocyte Adhesion Deficiency Using a SIN Lentiviral Vector with the PGK Promoter Expressing Canine CD18

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 219 KB

english, 2010

1138

527. Development of a Bioengineered Recombinant Factor VIII Therapeutic Using a Lentiviral Production Platform

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 219 KB

english, 2010

1139

533. Atomic Force Microscopy (AFM) Shows Lower Membrane Resistance of Human T Cells Rendered Deficient in WASP by RNAi

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 207 KB

english, 2010

1140

535. In Vivo Expansion of Regulatory T Cells with IL2-mAb Complexes Prevents and Modulates Anti-Factor VIII Immune Responses in Hemophilia A Mice after Factor VIII Plasmid-Mediated Gene Therapy

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 327 KB

english, 2010

1141

542. The Innate Immune Response to Intravenously Injected Adenoviral Vectors: Role of Endosomal Escape in MAP Kinase Activation and the Cytokine/Chemokine Response

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 284 KB

english, 2010

1142

543. AAV8 Vectors Evade CTL Mediated Destruction by Failing To Activate a Potent Inflammatory Response

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

1143

570. Development of a MDCK Cell Line for the Manufacturing of Canine Adenovirus Type 2 (CAV-2) Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 484 KB

english, 2010

1144

576. An Assay To Monitor the Residual Contamination in the Plasmid DNA Manufacturing Area Based on the Correlation of Plasmid DNA Concentration to TOC

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 361 KB

english, 2010

1145

580. Irradiation of Adult mdx Mice Prior to Full-Length Murine Dystrophin cDNA Transfer Results in a Delayed and Diminished Anti-Dystrophin Host Immune Response

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

1146

602. RNAi-Mediated Knockdown of HMG CoA Reductase In Vitro and In Vivo Increases Transgene Expression from Genomic LDLR Mini-Gene Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

1147

607. The Type III Interferon IL-28 Mediates Anti Tumor Efficacy of Oncolytic VSV in a Fully Immune Competent Model

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1148

608. A New Oncolytic Herpes Simplex Virus That Synergizes with Small Molecule Inhibitors of PI3K/Akt To Treat Malignant Glioma

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1149

612. Polyclonal Reconstitution of the Hematopoietic System in Two Patients of a WAS Clinical Gene Therapy Trial

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 198 KB

english, 2010

1150

620. Generation of a Transgenic Gut-Corrected CFTR Knockout Ferret Model with Intestinal-Specific Expression of Ferret CFTR

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 241 KB

english, 2010

1151

638. Novel Class of Adenovirus Vector “Capsid-Displaying” the Retro-Oriented Human Complement Inhibitor DAF Has a Significantly Improved Safety Profile In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 380 KB

english, 2010

1152

643. CAdVax-Vectored Multivalent Filovirus Vaccine Protects NHP Against Challenge at 1000 Times the Lethal Dose of Ebola and Marburg Viruses

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 267 KB

english, 2010

1153

650. Low Levels of CXCR4 Are Sufficient for HIV-1 Entry and Infection

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 229 KB

english, 2010

1154

658. Treatment of Cancer Patients with Integrin-Targeted Oncolytic Adenoviruses Ad5-D24-RGD and Ad5-RGD-D24-GMCSF

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 227 KB

english, 2010

1155

673. A Novel Approach to Developing Recombinant AAV Producing Cell Lines

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 232 KB

english, 2010

1156

684. Insights on the Mechanisms of Measles Virus Entry into Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1157

686. In Vivo Immunization with Integration Deficient Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 255 KB

english, 2010

1158

718. Enhanced Transduction and Replication of Fiber-Modified Adenovirus in Murine Primary T Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

1159

719. Cloning of the Human Herpesvirus-6A (HHV-6A) Genome into the Bacterial Artificial Chromosomes (BACs) towards the Creation of Helper-Less Amplicon Vectors for Potential Use in Lymphocyte Gene Therapy and Vaccination

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 226 KB

english, 2010

1160

726. Transcriptional Repression of Transgenes Delivered by Sleeping Beauty, piggyBac and Tol2 DNA Transposon Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 228 KB

english, 2010

1161

731. A Combinatorial Approach for Targeted, Systemic Delivery Using Small Molecules and Reversible Masking to Bypass Non-Specific Uptake In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 230 KB

english, 2010

1162

734. A Liver-Directed Targeting Tether Vector System To Increase Uptake in Liver Cells

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 193 KB

english, 2010

1163

737. Development of Post-Translational Tumor-Targeted-Cytokine Vector for Systemic Treatment of Malignant Disease

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 299 KB

english, 2010

1164

745. A Novel Cationic Lipophosphoramide with Di-Unsaturated Lipid Chains: Synthesis, Physico-Chemical Properties, and Transfection Activities

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 237 KB

english, 2010

1165

753. Bisphosphonate-Mediated Gene-Eluting Metal Stent as Gene Vector Delivery Using Antibody for Localized Gene Delivery

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

1166

768. Effects of Neonatal Intracranial AAV and Systemic Lentiviral Gene Therapy in Sanfilippo Syndrome Type B Mice

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 235 KB

english, 2010

1167

778. Primate Hippocampal Gene Delivery Via Frameless Stereotaxis

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 257 KB

english, 2010

1168

780. Modification of the Inhibitory Environment of Spinal Cord Injury by Lentiviral Vector Containing NT-3 and shRNA for NG2

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1169

785. High Force Myosin Correlated with the Early Degeneration of Jaw Closing Muscle in the GRMD Model of DMD

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 208 KB

english, 2010

1170

791. A Comparison of Adeno-Associated Virus Mediated Gene Transfer into Healthy and Dystrophin-Deficient Skeletal Muscles

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

1171

792. In Vivo Reprogramming into the Myogenic Lineage and Improving Engraftment in the mdx Mouse Model of DMD

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

1172

809. Time for TEA: T Cells Expanded on Artificial Antigen Presenting Cells?

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 340 KB

english, 2010

1173

815. Genetically Mediated Constitutive Expression of CD40L by T Cells Will Induce Enhanced Immunogenicity of B-Cell Malignancies

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 238 KB

english, 2010

1174

819. Autocrine, Not Paracrine, Interferon-Gamma Gene Delivery Enhances Ex Vivo Antigen-Specific Cytotoxic T Lymphocyte Stimulation and Killing

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 501 KB

english, 2010

1175

825. Small Plasmid/PEI/Anionic Polysaccharide Ternary Complex Particles: Toxicity and Animal Clinical Study

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 250 KB

english, 2010

1176

836. Armed and Transcriptionally Driven Oncolytic HSV-1 Enhances Antitumor Efficacy In Vitro and In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 258 KB

english, 2010

1177

841. Enhanced Tumor Killing by a Mortalin Targeting Modified Oncolytic Adenovirus

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 239 KB

english, 2010

1178

857. High Incidence of Vector Integration near Cancer-Related Genes within Primitive Hematopoietic Stem Cells (HSC) after Fetal Gene Transfer with γ-Retroviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 312 KB

english, 2010

1179

861. Transduction of Human CD34+ Derived NOG Repopulating Cells with an Insulated SIN Lentiviral Vector for SCID-X1 Produced from a Stable Producer Cell Line

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 209 KB

english, 2010

1180

870. Delivery of Soluble ICAM-1 in NOD Salivary Glands Can Result in B Cell Proliferation and Immunoglobulin Production

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 240 KB

english, 2010

1181

872. Efficient Gene Delivery to Pig Airways Using Helper-Dependent Adenoviral Vectors

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 259 KB

english, 2010

1182

880. Repeat Aerosol Delivery of Concentrated PEI/pDNA to the Sheep Lung

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 287 KB

english, 2010

1183

886. Design and Pre-Clinical Evaluation of Adeno-Associated Viral (AAV) Vectors for Therapeutic Inhibition of Disease-Associated miRNAs

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 286 KB

english, 2010

1184

887. Safety Results of a Phase 1 Clinical Study for Treatment of Patients with Advanced Solid Tumors with the TLR9 Agonist MGN1703

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

1185

890. Screening of Nature Compounds for Enhancing Antisense Oligonucleotide-Induced Exon Skipping of Dystrophin Gene In Vitro and In Vivo

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 247 KB

english, 2010

1186

897. Gene Editing Activity in C. elegans Directed by Single-Stranded Oligonucleotides

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 285 KB

english, 2010

1187

Corrigendum to “Enhanced Antitumor Effects of an Engineered Measles Virus Edmonston Strain Expressing the Wild-type N, P, L Genes on Human Renal Cell Carcinoma”

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 44 KB

english, 2010

1188

Corrigendum to “Coadministration of Telomerase Genetic Vaccine and a Novel TLR9 Agonist in Nonhuman Primates”

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 73 KB

english, 2010

1189

In the Beginning: Reflections on the Genesis of Molecular Therapy

Wilson, James M

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 954 KB

english, 2010

1190

Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and γc Overexpression

Ginn, Samantha L, Liao, Sophia HY, Dane, Allison P, Hu, Min, Hyman, Jessica, Finnie, John W, Zheng, Maolin, Cavazzana-Calvo, Marina, Alexander, Stephen I, Thrasher, Adrian J, Alexander, Ian E

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.54 MB

english, 2010

1191

Corrigendum to “Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood-Brain Barrier (BBB)”

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 36 KB

2010

1192

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 993 KB

english, 2010

1193

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.15 MB

english, 2010

1194

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 630 KB

english, 2010

1195

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.38 MB

english, 2010

1196

AUTHOR INDEX

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 320 KB

2010

1197

American Society of Gene and Cell Therapy 13th Annual Meeting

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.12 MB

english, 2010

1198

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 565 KB

english, 2010

1199

Interesting Times: Molecular Therapy and the Evolving Media of Scientific Publishing

Brenner, Malcolm

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 930 KB

english, 2010

1200

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 641 KB

english, 2010

1201

In This Issue

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 1.12 MB

2010

1202

Low Current-driven Micro-electroporation Allows Efficient In Vivo Delivery of Nonviral DNA into the Adult Mouse Brain

Vry, Jochen De, Martínez-Martínez, Pilar, Losen, Mario, Bode, Gerard H, Temel, Yasin, Steckler, Thomas, Steinbusch, Harry WM, Baets, Marc De, Prickaerts, Jos

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 2.03 MB

english, 2010

1203

Corrigendum to “Recent Advances in Lentiviral Vector Development and Applications”

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 42 KB

2010

1204

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 505 KB

english, 2010

1205

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.12 MB

english, 2010

1206

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.18 MB

english, 2010

1207

Corrigendum to “Therapeutic and Tumor-specific Immunity Induced by Combination of Dendritic Cells and Oncolytic Adenovirus Expressing IL-12 and 4-1BBL”

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 34 KB

2010

1208

Corrigendum to “Gene Therapy of Mpl Deficiency: Challenging Balance Between Leukemia and Pancytopenia”

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 46 KB

english, 2010

1209

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.09 MB

english, 2010

1210

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 687 KB

english, 2010

1211

Corrigendum to “Gene Transfer Efficiency and Genome-Wide Integration Profiling of Sleeping Beauty, Tol2, and PiggyBac Transposons in Human Primary T Cells”

Journal:

Molecular Therapy

Year:

2010

File:

PDF, 39 KB

2010

1212

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 428 KB

english, 2010

1213

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 457 KB

english, 2010

1214

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 533 KB

english, 2010

1215

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.19 MB

english, 2010

1216

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.16 MB

english, 2010

1217

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.03 MB

english, 2010

1218

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 540 KB

english, 2010

1219

In This Issue

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 1.13 MB

english, 2010

1220

Introducing eJP

Frederickson, Robert M, Brenner, Malcolm K

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 484 KB

english, 2010

1221

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 563 KB

english, 2010

1222

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 681 KB

english, 2010

1223

Research Highlights

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 606 KB

english, 2010

1224

KEYWORD INDEX

Journal:

Molecular Therapy

Year:

2010

Language:

english

File:

PDF, 268 KB

english, 2010

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