Volume 20

Molecular Therapy

Volume 20

1

Advances in Cell and Gene-based Therapies for Cystic Fibrosis Lung Disease

Oakland, Mayumi, Sinn, Patrick L, McCray Jr, Paul B

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 795 KB

english, 2012

2

Cell-penetrating Peptides as Versatile Vehicles for Oligonucleotide Delivery

Margus, Helerin, Padari, Kärt, Pooga, Margus

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 218 KB

english, 2012

3

Extensive Methylation of Promoter Sequences Silences Lentiviral Transgene Expression During Stem Cell Differentiation In Vivo

Herbst, Friederike, Ball, Claudia R, Tuorto, Francesca, Nowrouzi, Ali, Wang, Wei, Zavidij, Oksana, Dieter, Sebastian M, Fessler, Sylvia, van der Hoeven, Franciscus, Kloz, Ulrich

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 400 KB

english, 2012

4

Spherical Bullet Formation via E-cadherin Promotes Therapeutic Potency of Mesenchymal Stem Cells Derived From Human Umbilical Cord Blood for Myocardial Infarction

Lee, Eun Ju, Park, Sung Jung, Kang, Soo Kyoung, Kim, Gi-Hwan, Kang, Hyun-Jae, Lee, Sae-Won, Jeon, Hong Bae, Kim, Hyo-Soo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 3.11 MB

english, 2012

5

Hepatitis C Virus NS3/NS4A DNA Vaccine Induces Multiepitope T Cell Responses in Rhesus Macaques Mimicking Human Immune Responses

Lang Kuhs, Krystle A, Ginsberg, Arielle A, Yan, Jian, Wiseman, Roger W, Khan, Amir S, Sardesai, Niranjan Y, O'Connor, David H, Weiner, David B

Journal:

Molecular Therapy

Year:

2011

Language:

english

File:

PDF, 641 KB

english, 2011

6

Progress Toward In Vivo Use of siRNAs-II

Rettig, Garrett R, Behlke, Mark A

Journal:

Molecular Therapy

Year:

2011

Language:

english

File:

PDF, 368 KB

english, 2011

7

Action and Reaction: The Biological Response to siRNA and Its Delivery Vehicles

Kanasty, Rosemary L, Whitehead, Kathryn A, Vegas, Arturo J, Anderson, Daniel G

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 745 KB

english, 2012

8

Treating Tumors With a Vaccinia Virus Expressing IFNβ Illustrates the Complex Relationships Between Oncolytic Ability and Immunogenicity

Wang, Liang-Chuan S, Lynn, Rachel C, Cheng, Guanjun, Alexander, Edward, Kapoor, Veena, Moon, Edmund K, Sun, Jing, Fridlender, Zvi G, Isaacs, Stuart N, Thorne, Stephen H

Journal:

Molecular Therapy

Year:

2011

Language:

english

File:

PDF, 650 KB

english, 2011

9

In Vivo Gene Delivery by Nonviral Vectors: Overcoming Hurdles?

Zhang, Yuan, Satterlee, Andrew, Huang, Leaf

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 262 KB

english, 2012

10

Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies

Wang, Zejing, Storb, Rainer, Halbert, Christine L, Banks, Glen B., Butts, Tiffany M., Finn, Eric E., Allen, James M., Miller, A. Dusty, Chamberlain, Jeffrey S., Tapscott, Stephen J.

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.67 MB

english, 2012

11

The β-Globin Locus Control Region in Combination With the EF1α Short Promoter Allows Enhanced Lentiviral Vector-mediated Erythroid Gene Expression With Conserved Multilineage Activity

Montiel-Equihua, Claudia A, Zhang, Lin, Knight, Sean, Saadeh, Heba, Scholz, Simone, Carmo, Marlene, Alonso-Ferrero, Maria E, Blundell, Michael P, Monkeviciute, Aiste, Schulz, Reiner, Collins, Mary, Ta

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 265 KB

english, 2012

12

Clinical Development of Advanced Therapy Medicinal Products in Europe: Evidence That Regulators Must Be Proactive

Maciulaitis, Romaldas, D’Apote, Lucia, Buchanan, Andrew, Pioppo, Laura, Schneider, Christian K

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 508 KB

english, 2012

13

Phage Display-directed Discovery of LEDGF/p75 Binding Cyclic Peptide Inhibitors of HIV Replication

Desimmie, Belete A, Humbert, Michael, Lescrinier, Eveline, Hendrix, Jelle, Vets, Sofie, Gijsbers, Rik, Ruprecht, Ruth M, Dietrich, Ursula, Debyser, Zeger, Christ, Frauke

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.54 MB

english, 2012

14

Adenovirus i-Leader Truncation Bioselected Against Cancer-associated Fibroblasts to Overcome Tumor Stromal Barriers

Puig-Saus, Cristina, Gros, Alena, Alemany, Ramon, Cascalló, Manel

Journal:

Molecular Therapy

Year:

2011

Language:

english

File:

PDF, 446 KB

english, 2011

15

Regulation of Stem Cell Plasticity: Mechanisms and Relevance to Tissue Biology and Cancer

Hamerlik, Petra, Lieber, André, Bartek, Jiri, Strauss, Robert

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 247 KB

english, 2012

16

Immune-mediated Loss of Transgene Expression From Virally Transduced Brain Cells Is Irreversible, Mediated by IFNγ, Perforin, and TNFα, and due to the Elimination of Transduced Cells

Zirger, Jeffrey M, Puntel, Mariana, Bergeron, Josee, Wibowo, Mia, Moridzadeh, Rameen, Bondale, Niyati, Barcia, Carlos, Kroeger, Kurt M, Liu, Chunyan, Castro, Maria G

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.42 MB

english, 2012

17

A TLR and Non-TLR Mediated Innate Response to Lentiviruses Restricts Hepatocyte Entry and Can be Ameliorated by Pharmacological Blockade

Agudo, Judith, Ruzo, Albert, Kitur, Kipyegon, Sachidanandam, Ravi, Blander, J Magarian, Brown, Brian D

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.92 MB

english, 2012

18

N-cadherin Determines Individual Variations in the Therapeutic Efficacy of Human Umbilical Cord Blood-derived Mesenchymal Stem Cells in a Rat Model of Myocardial Infarction

Lee, Eun Ju, Choi, Eue-Keun, Kang, Soo Kyoung, Kim, Gi-Hwan, Park, Ju Young, Kang, Hyun-Jae, Lee, Sae-Won, Kim, Keum-Hyun, Kwon, Jin Sook, Lee, Ki Hong

Journal:

Molecular Therapy

Year:

2011

Language:

english

File:

PDF, 1.18 MB

english, 2011

19

Treatment of Chemotherapy-Refractory Cancer in the Advanced Therapy Access Program

Hemminki, Akseli

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 178 KB

english, 2012

20

Robust Suppression of HIV Replication by Intracellularly Expressed Reverse Transcriptase Aptamers Is Independent of Ribozyme Processing

Lange, Margaret J, Sharma, Tarun K, Whatley, Angela S, Landon, Linda A, Tempesta, Michael A, Johnson, Marc C, Burke, Donald H

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.69 MB

english, 2012

21

The Need for Increased Clarity and Transparency in the Regulatory Pathway for Gene Medicines in the European Union

Ylä-Herttuala, Seppo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

22

Radiation-inducible Immunotherapy for Cancer: Senescent Tumor Cells as a Cancer Vaccine

Meng, Yuru, Efimova, Elena V, Hamzeh, Khaled W, Darga, Thomas E, Mauceri, Helena J., Fu, Yang-Xin, Kron, Stephen J, Weichselbaum, Ralph R

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 3.21 MB

english, 2012

23

Dendritic Cells Loaded With mRNA Encoding Full-length Tumor Antigens Prime CD4+ and CD8+ T Cells in Melanoma Patients

Van Nuffel, An MT, Benteyn, Daphné, Wilgenhof, Sofie, Pierret, Lauranne, Corthals, Jurgen, Heirman, Carlo, van der Bruggen, Pierre, Coulie, Pierre G, Neyns, Bart, Thielemans, Kris

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.70 MB

english, 2012

24

Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

Vulin, Adeline, Barthélémy, Inès, Goyenvalle, Aurélie, Thibaud, Jean-Laurent, Beley, Cyriaque, Griffith, Graziella, Benchaouir, Rachid, le Hir, Maëva, Unterfinger, Yves, Lorain, Stéphanie, Dreyfus, Pa

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.97 MB

english, 2012

25

Long-term Restoration of Cardiac Dystrophin Expression in Golden Retriever Muscular Dystrophy Following rAAV6-mediated Exon Skipping

Bish, Lawrence T, Sleeper, Meg M, Forbes, Sean C, Wang, Bingjing, Reynolds, Caryn, Singletary, Gretchen E, Trafny, Dennis, Morine, Kevin J, Sanmiguel, Julio, Cecchini, Sylvain

Journal:

Molecular Therapy

Year:

2011

Language:

english

File:

PDF, 452 KB

english, 2011

26

Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union

Ylä-Herttuala, Seppo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 338 KB

english, 2012

27

SCL/TAL1 Regulates Hematopoietic Specification From Human Embryonic Stem Cells

Real, Pedro J, Ligero, Gertrudis, Ayllon, Veronica, Ramos-Mejia, Veronica, Bueno, Clara, Gutierrez-Aranda, Ivan, Navarro-Montero, Oscar, Lako, Majlinda, Menendez, Pablo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 369 KB

english, 2012

28

Efficient Generation of Functional Hepatocytes From Human Embryonic Stem Cells and Induced Pluripotent Stem Cells by HNF4α Transduction

Takayama, Kazuo, Inamura, Mitsuru, Kawabata, Kenji, Katayama, Kazufumi, Higuchi, Maiko, Tashiro, Katsuhisa, Nonaka, Aki, Sakurai, Fuminori, Hayakawa, Takao, Kusuda Furue, Miho

Journal:

Molecular Therapy

Year:

2011

Language:

english

File:

PDF, 699 KB

english, 2011

29

In Vivo Delivery of Nucleic Acids via Glycopolymer Vehicles Affords Therapeutic Infarct Size Reduction In Vivo

Tranter, Michael, Liu, Yemin, He, Suiwen, Gulick, James, Ren, Xiaoping, Robbins, Jeffrey, Jones, W. Keith, Reineke, Theresa M.

Journal:

Molecular Therapy

Year:

2011

Language:

english

File:

PDF, 462 KB

english, 2011

30

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Bowles, Dawn E, McPhee, Scott WJ, Li, Chengwen, Gray, Steven J, Samulski, Jade J, Camp, Angelique S, Li, Juan, Wang, Bing, Monahan, Paul E, Rabinowitz, Joseph E

Journal:

Molecular Therapy

Year:

2011

Language:

english

File:

PDF, 688 KB

english, 2011

31

ChAd63-MVA–vectored Blood-stage Malaria Vaccines Targeting MSP1 and AMA1: Assessment of Efficacy Against Mosquito Bite Challenge in Humans

Sheehy, Susanne H, Duncan, Christopher JA, Elias, Sean C, Choudhary, Prateek, Biswas, Sumi, Halstead, Fenella D, Collins, Katharine A, Edwards, Nick J, Douglas, Alexander D, Anagnostou, Nicholas A, Ew

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.13 MB

english, 2012

32

Oncolytic Sindbis Virus Targets Tumors Defective in the Interferon Response and Induces Significant Bystander Antitumor Immunity In Vivo

Huang, Pong-Yu, Guo, Jih-Huong, Hwang, Lih-Hwa

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 668 KB

english, 2012

33

Inhibition of Hepatitis C Virus Replication by Intracellular Delivery of Multiple siRNAs by Nanosomes

Chandra, Partha K, Kundu, Anup K, Hazari, Sidhartha, Chandra, Sruti, Bao, Lili, Ooms, Tara, Morris, Gilbert F, Wu, Tong, Mandal, Tarun K, Dash, Srikanta

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 6.46 MB

english, 2012

34

Preclinical Corrective Gene Transfer in Xeroderma Pigmentosum Human Skin Stem Cells

Warrick, Emilie, Garcia, Marta, Chagnoleau, Corinne, Chevallier, Odile, Bergoglio, Valérie, Sartori, Daniela, Mavilio, Fulvio, Angulo, Jaime F, Avril, Marie-Françoise, Sarasin, Alain, Larcher, Fernand

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 772 KB

english, 2012

35

PEGylated Polyplex With Optimized PEG Shielding Enhances Gene Introduction in Lungs by Minimizing Inflammatory Responses

Uchida, Satoshi, Itaka, Keiji, Chen, Qixian, Osada, Kensuke, Ishii, Takehiko, Shibata, Masa-Aki, Harada-Shiba, Mariko, Kataoka, Kazunori

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 681 KB

english, 2012

36

Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

Cirak, Sebahattin, Feng, Lucy, Anthony, Karen, Arechavala-Gomeza, Virginia, Torelli, Silvia, Sewry, Caroline, Morgan, Jennifer E, Muntoni, Francesco

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 350 KB

english, 2012

37

RNAi Targeting CXCR4 Inhibits Tumor Growth Through Inducing Cell Cycle Arrest and Apoptosis

Yu, Tao, Wu, Yingying, Huang, Yi, Yan, Chaoran, Liu, Ying, Wang, Zongsheng, Wang, Xiaoyi, Wen, Yuming, Wang, Changmei, Li, Longjiang

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 797 KB

english, 2012

38

Failure of Translation of Human Adenovirus mRNA in Murine Cancer Cells Can be Partially Overcome by L4-100K Expression In Vitro and In Vivo

Young, Anna-Mary, Archibald, Kyra M, Tookman, Laura A, Pool, Alexander, Dudek, Kate, Jones, Carolyn, Williams, Sarah L, Pirlo, Katrina J, Willis, Anne E, Lockley, Michelle, McNeish, Iain A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.82 MB

english, 2012

39

Adenovirus Immunity: X Marks the Spot

Suzuki, Masataka, Rooney, Cliona M

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 669 KB

english, 2012

40

Human Erythropoietin Gene Delivery Using an Arginine-grafted Bioreducible Polymer System

Lee, Youngsook, Nam, Hye Yeong, Kim, Jaesung, Lee, Minhyung, Yockman, James W, Shin, Sug Kyun, Kim, Sung Wan

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 305 KB

english, 2012

41

Still Seeking an Effective “One–Two” Malaria Vaccine Punch

Smith, Joseph D, Sack, Brandon K

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 775 KB

english, 2012

42

Proinflammatory Macrophages Enhance the Regenerative Capacity of Human Myoblasts by Modifying Their Kinetics of Proliferation and Differentiation

Bencze, Maximilien, Negroni, Elisa, Vallese, Denis, Yacoub–Youssef, Houda, Chaouch, Soraya, Wolff, Annie, Aamiri, Ahmed, Di Santo, James P, Chazaud, Bénédicte, Butler-Browne, Gillian, Savino, Wilson,

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 5.77 MB

english, 2012

43

A Tubulin Binding Peptide Targets Glioma Cells Disrupting Their Microtubules, Blocking Migration, and Inducing Apoptosis

Berges, Raphael, Balzeau, Julien, Peterson, Alan C, Eyer, Joel

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 782 KB

english, 2012

44

Directed Evolution of Adeno-associated Virus for Enhanced Gene Delivery and Gene Targeting in Human Pluripotent Stem Cells

Asuri, Prashanth, Bartel, Melissa A, Vazin, Tandis, Jang, Jae-Hyung, Wong, Tiffany B, Schaffer, David V

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 501 KB

english, 2012

45

A General RNA Motif for Cellular Transfection

Magalhães, Maria LB, Byrom, Michelle, Yan, Amy, Kelly, Linsley, Li, Na, Furtado, Raquel, Palliser, Deborah, Ellington, Andrew D, Levy, Matthew

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 786 KB

english, 2012

46

Immunotherapeutic Synergy Between Anti-CD137 mAb and Intratumoral Administration of a Cytopathic Semliki Forest Virus Encoding IL-12

Quetglas, José I, Dubrot, Juan, Bezunartea, Jaione, Sanmamed, Miguel F, Hervas-Stubbs, Sandra, Smerdou, Cristian, Melero, Ignacio

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.85 MB

english, 2012

47

An Oncolytic Adenovirus Enhanced for Toll-like Receptor 9 Stimulation Increases Antitumor Immune Responses and Tumor Clearance

Cerullo, Vincenzo, Diaconu, Iulia, Romano, Valentina, Hirvinen, Mari, Ugolini, Matteo, Escutenaire, Sophie, Holm, Sirkka-Liisa, Kipar, Anja, Kanerva, Anna, Hemminki, Akseli

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 606 KB

english, 2012

48

Rad51 Promoter-Targeted Gene Therapy Is Effective for In Vivo Visualization and Treatment of Cancer

Hine, Christopher M, Seluanov, Andrei, Gorbunova, Vera

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1022 KB

english, 2012

49

Interferon-β Sensitivity of Tumor Cells Correlates With Poor Response to VA7 Virotherapy in Mouse Glioma Models

Ruotsalainen, Janne, Martikainen, Miika, Niittykoski, Minna, Huhtala, Tuulia, Aaltonen, Tytti, Heikkilä, Jari, Bell, John, Vähä-Koskela, Markus, Hinkkanen, Ari

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 745 KB

english, 2012

50

Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy

Goyenvalle, Aurélie, Wright, Jordan, Babbs, Arran, Wilkins, Vivienne, Garcia, Luis, Davies, Kay E

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 376 KB

english, 2012

51

Zinc-finger Nuclease Editing of Human cxcr4 Promotes HIV-1 CD4+ T Cell Resistance and Enrichment

Yuan, Jinyun, Wang, Jianbin, Crain, Karen, Fearns, Colleen, Kim, Kenneth A, Hua, Kevin L, Gregory, Philip D, Holmes, Michael C, Torbett, Bruce E

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 525 KB

english, 2012

52

Targeting Axl With an High-affinity Inhibitory Aptamer

Cerchia, Laura, Esposito, Carla L, Camorani, Simona, Rienzo, Anna, Stasio, Loredana, Insabato, Luigi, Affuso, Andrea, de Franciscis, Vittorio

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 930 KB

english, 2012

53

Athletes’ Use of Unproven Stem Cell Therapies: Adding to Inappropriate Media Hype?

Caulfield, Timothy, McGuire, Amy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.31 MB

english, 2012

54

Phase I Clinical Trial of Smad7 Knockdown Using Antisense Oligonucleotide in Patients With Active Crohn's Disease

Monteleone, Giovanni, Fantini, Massimo C, Onali, Sara, Zorzi, Francesca, Sancesario, Giulia, Bernardini, Sergio, Calabrese, Emma, Viti, Francesca, Monteleone, Ivan, Biancone, Livia, Pallone, Francesco

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 385 KB

english, 2012

55

DNA Vaccination in the Skin Using Microneedles Improves Protection Against Influenza

Song, Jae-Min, Kim, Yeu-Chun, O, Eunju, Compans, Richard W, Prausnitz, Mark R, Kang, Sang-Moo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 350 KB

english, 2012

56

Mesenchymal Stromal Cells Treatment Attenuates Dry Eye in Patients With Chronic Graft-versus-host Disease

Weng, Jianyu, He, Chang, Lai, Peilong, Luo, Chenwei, Guo, Rong, Wu, Suijing, Geng, Suxia, Xiangpeng, Andy, Liu, Xialin, Du, Xin

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 859 KB

english, 2012

57

Chemoselection of Allogeneic HSC After Murine Neonatal Transplantation Without Myeloablation or Post-transplant Immunosuppression

Falahati, Rustom, Zhang, Jianqing, Flebbe-Rehwaldt, Linda, Shi, Yimin, Gerson, Stanton L, Gaensler, Karin ML

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.72 MB

english, 2012

58

Retargeting Sleeping Beauty Transposon Insertions by Engineered Zinc Finger DNA-binding Domains

Voigt, Katrin, Gogol-Döring, Andreas, Miskey, Csaba, Chen, Wei, Cathomen, Toni, Izsvák, Zsuzsanna, Ivics, Zoltán

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 850 KB

english, 2012

59

Terminal Differentiation of Cardiac and Skeletal Myocytes Induces Permissivity to AAV Transduction by Relieving Inhibition Imposed by DNA Damage Response Proteins

Lovric, Jasmina, Mano, Miguel, Zentilin, Lorena, Eulalio, Ana, Zacchigna, Serena, Giacca, Mauro

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 6.10 MB

english, 2012

60

Inflammation, Apoptosis, and Necrosis Induced by Neoadjuvant Fas Ligand Gene Therapy Improves Survival of Dogs With Spontaneous Bone Cancer

Modiano, Jaime F, Bellgrau, Donald, Cutter, Gary R, Lana, Susan E, Ehrhart, Nicole P, Ehrhart, EJ, Wilke, Vicki L, Charles, J Brad, Munson, Sibyl, Scott, Milcah C, Pozniak, John, Carlson, Cathy S, Sch

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 778 KB

english, 2012

61

A New Therapeutic Approach Using a Schizophyllan-based Drug Delivery System for Inflammatory Bowel Disease

Takedatsu, Hidetoshi, Mitsuyama, Keiichi, Mochizuki, Shinichi, Kobayashi, Teppei, Sakurai, Kazuo, Takeda, Hiroshi, Fujiyama, Yoshihide, Koyama, Yoshikazu, Nishihira, Jun, Sata, Michio

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 463 KB

english, 2012

62

Ad5:Ad48 Hexon Hypervariable Region Substitutions Lead to Toxicity and Increased Inflammatory Responses Following Intravenous Delivery

Coughlan, Lynda, Bradshaw, Angela C, Parker, Alan L, Robinson, Hollie, White, Katie, Custers, Jerome, Goudsmit, Jaap, Van Roijen, Nico, Barouch, Dan H, Nicklin, Stuart A, Baker, Andrew H

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.07 MB

english, 2012

63

Exon 45 Skipping Through U1-snRNA Antisense Molecules Recovers the Dys-nNOS Pathway and Muscle Differentiation in Human DMD Myoblasts

Cazzella, Valentina, Martone, Julie, Pinnarò, Chiara, Santini, Tiziana, Twayana, Shyam Sundar, Sthandier, Olga, D'Amico, Adele, Ricotti, Valeria, Bertini, Enrico, Muntoni, Francesco, Bozzoni, Irene

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.34 MB

english, 2012

64

A Tumor-stroma Targeted Oncolytic Adenovirus Replicated in Human Ovary Cancer Samples and Inhibited Growth of Disseminated Solid Tumors in Mice

Lopez, M Veronica, Rivera, Angel A, Viale, Diego L, Benedetti, Lorena, Cuneo, Nicasio, Kimball, Kristopher J, Wang, Minghui, Douglas, Joanne T, Zhu, Zeng B, Bravo, Alicia I, Gidekel, Manuel, Alvarez,

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 619 KB

english, 2012

65

HSV-1 Amplicon Vectors Launch the Production of Heterologous Rotavirus-like Particles and Induce Rotavirus-specific Immune Responses in Mice

Laimbacher, Andrea S, Esteban, Laura E, Castello, Alejandro A, Abdusetir Cerfoglio, Juan C, Argüelles, Marcelo H, Glikmann, Graciela, D'Antuono, Alejandra, Mattion, Nora, Berois, Mabel, Arbiza, Juan,

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 3.55 MB

english, 2012

66

RNA Interference Inhibits DUX4-induced Muscle Toxicity In Vivo: Implications for a Targeted FSHD Therapy

Wallace, Lindsay M, Liu, Jian, Domire, Jacqueline S, Garwick-Coppens, Sara E, Guckes, Susan M, Mendell, Jerry R, Flanigan, Kevin M, Harper, Scott Q.

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 534 KB

english, 2012

67

Lentivector Transduction Improves Outcomes Over Transplantation of Human HSCs Alone in NOD/SCID/Fabry Mice

Pacienza, Natalia, Yoshimitsu, Makoto, Mizue, Nobuo, Au, Bryan CY, Wang, James CM, Fan, Xin, Takenaka, Toshihiro, Medin, Jeffrey A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 527 KB

english, 2012

68

miR-29 Inhibits Bleomycin-induced Pulmonary Fibrosis in Mice

Xiao, Jun, Meng, Xiao-Ming, Huang, Xiao R, Chung, Arthur CK, Feng, Yu-Lin, Hui, David SC, Yu, Cheuk-Man, Sung, Joseph JY, Lan, Hui Y

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.92 MB

english, 2012

69

Efficient Reprogramming of Human Cord Blood CD34+ Cells Into Induced Pluripotent Stem Cells With OCT4 and SOX2 Alone

Meng, Xianmei, Neises, Amanda, Su, Rui-Jun, Payne, Kimberly J, Ritter, Linda, Gridley, Daila S, Wang, Jun, Sheng, Matilda, William Lau, K-H, Baylink, David J, Zhang, Xiao-Bing

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 529 KB

english, 2012

70

The Extragenic Spacer Length Between the 5′ and 3′ Ends of the Transgene Expression Cassette Affects Transgene Silencing From Plasmid-based Vectors

Lu, Jiamiao, Zhang, Feijie, Xu, Siqun, Fire, Andrew Z, Kay, Mark A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 874 KB

english, 2012

71

CNS-specific Therapy for Ongoing EAE by Silencing IL-17 Pathway in Astrocytes

Yan, Yaping, Ding, Xiaoli, Li, Ke, Ciric, Bogoljub, Wu, Shuai, Xu, Hui, Gran, Bruno, Rostami, Abdolmohamad, Zhang, Guang-Xian

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 683 KB

english, 2012

72

AAV9-mediated VEGF-B Gene Transfer Improves Systolic Function in Progressive Left Ventricular Hypertrophy

Huusko, Jenni, Lottonen, Line, Merentie, Mari, Gurzeler, Erika, Anisimov, Andrey, Miyanohara, Atsushi, Alitalo, Kari, Tavi, Pasi, Ylä-Herttuala, Seppo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.55 MB

english, 2012

73

Incorporation of the B18R Gene of Vaccinia Virus Into an Oncolytic Herpes Simplex Virus Improves Antitumor Activity

Fu, Xinping, Rivera, Armando, Tao, Lihua, Zhang, Xiaoliu

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 606 KB

english, 2012

74

GOLGA2/GM130, cis-Golgi Matrix Protein, is a Novel Target of Anticancer Gene Therapy

Chang, Seung-Hee, Hong, Seong-Ho, Jiang, Hu-Lin, Minai-Tehrani, Arash, Yu, Kyeong-Nam, Lee, Jae-Ho, Kim, Ji-Eun, Shin, Ji-Young, Kang, Bitna, Park, Sungjin, Han, Kiwon, Chae, Chanhee, Cho, Myung-Haing

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 7.05 MB

english, 2012

75

Isolation and Optimization of Murine IL-10 Receptor Blocking Oligonucleotide Aptamers Using High-throughput Sequencing

Berezhnoy, Alexey, Stewart, C. Andrew, Mcnamara II, James O., Thiel, William, Giangrande, Paloma, Trinchieri, Giorgio, Gilboa, Eli

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.01 MB

english, 2012

76

Modulation of eIF5A Expression Using SNS01 Nanoparticles Inhibits NF-κB Activity and Tumor Growth in Murine Models of Multiple Myeloma

Taylor, Catherine A, Liu, Zhongda, Tang, Terence C, Zheng, Qifa, Francis, Sarah, Wang, Tzann-Wei, Ye, Bin, Lust, John A, Dondero, Richard, Thompson, John E

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 419 KB

english, 2012

77

Myxoma Virus Sensitizes Cancer Cells to Gemcitabine and Is an Effective Oncolytic Virotherapeutic in Models of Disseminated Pancreatic Cancer

Wennier, Sonia Tusell, Liu, Jia, Li, Shoudong, Rahman, Masmudur M., Mona, Mahmoud, McFadden, Grant

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 879 KB

english, 2012

78

RNAi Joins the “Singles Club”

Peschansky, Veronica J., Liang, Zicai, Wahlestedt, Claes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 755 KB

english, 2012

79

Patterns of scAAV Vector Insertion Associated With Oncogenic Events in a Mouse Model for Genotoxicity

Rosas, Lucia E, Grieves, Jessica L, Zaraspe, Kimberly, La Perle, Krista MD, Fu, Haiyan, McCarty, Douglas M

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 690 KB

english, 2012

80

Harnessing Oncolytic Virus-mediated Antitumor Immunity in an Infected Cell Vaccine

Lemay, Chantal G, Rintoul, Julia L, Kus, Agnieszka, Paterson, Jennifer M, Garcia, Vanessa, Falls, Theresa J, Ferreira, Lisa, Bridle, Byram W, Conrad, David P, Tang, Vera A, Diallo, Jean-Simon, Arulana

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.48 MB

english, 2012

81

Mixed Vector Immunization With Recombinant Adenovirus and MVA Can Improve Vaccine Efficacy While Decreasing Antivector Immunity

Reyes-Sandoval, Arturo, Rollier, Christine S, Milicic, Anita, Bauza, Karolis, Cottingham, Matthew G, Tang, Choon-Kit, Dicks, Matthew D, Wang, Dong, Longley, Rhea J, Wyllie, David H, Hill, Adrian VS

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 775 KB

english, 2012

82

Oral Administration of Recombinant Adeno-associated Virus-mediated Bone Morphogenetic Protein-7 Suppresses CCl4-induced Hepatic Fibrosis in Mice

Hao, Zhi-Ming, Cai, Min, Lv, Yi-Fei, Huang, Yan-Hua, Li, Hong-Hong

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.61 MB

english, 2012

83

A CRISPR Approach to Gene Targeting

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.31 MB

english, 2012

84

Microvesicle-associated AAV Vector as a Novel Gene Delivery System

Maguire, Casey A, Balaj, Leonora, Sivaraman, Sarada, Crommentuijn, Matheus HW, Ericsson, Maria, Mincheva-Nilsson, Lucia, Baranov, Vladimir, Gianni, Davide, Tannous, Bakhos A, Sena-Esteves, Miguel, Bre

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.08 MB

english, 2012

85

Intravenous Mesenchymal Stem Cells Prevented Rejection of Allogeneic Corneal Transplants by Aborting the Early Inflammatory Response

Oh, Joo Youn, Lee, Ryang Hwa, Yu, Ji Min, Ko, Jung Hwa, Lee, Hyun Ju, Ko, Ah Young, Roddy, Gavin W, Prockop, Darwin J

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.47 MB

english, 2012

86

Cellular Therapy With Ixmyelocel-T to Treat Critical Limb Ischemia: The Randomized, Double-blind, Placebo-controlled RESTORE-CLI Trial

Powell, Richard J, Marston, William A, Berceli, Scott A, Guzman, Raul, Henry, Timothy D, Longcore, Amy T, Stern, Theresa P, Watling, Sharon, Bartel, Ronnda L

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

87

Valproic Acid Confers Functional Pluripotency to Human Amniotic Fluid Stem Cells in a Transgene-free Approach

Moschidou, Dafni, Mukherjee, Sayandip, Blundell, Michael P, Drews, Katharina, Jones, Gemma N, Abdulrazzak, Hassan, Nowakowska, Beata, Phoolchund, Anju, Lay, Kenneth, Ramasamy, T Selvee, Cananzi, Mara,

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.47 MB

english, 2012

88

Reversal of Diabetes Through Gene Therapy of Diabetic Rats by Hepatic Insulin Expression via Lentiviral Transduction

Elsner, Matthias, Terbish, Taivankhuu, Jörns, Anne, Naujok, Ortwin, Wedekind, Dirk, Hedrich, Hans-Jürgen, Lenzen, Sigurd

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 434 KB

english, 2012

89

In Vivo Tracking of Mesechymal Stem Cells Using Fluorescent Nanoparticles in an Osteochondral Repair Model

Lee, Jong Min, Kim, Byung-Soo, Lee, Haeshin, Im, Gun-Il

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 580 KB

english, 2012

90

Efficient Gene Therapy for Parkinson's Disease Using Astrocytes as Hosts for Localized Neurotrophic Factor Delivery

Drinkut, Anja, Tereshchenko, Yuliya, Schulz, Jörg B, Bähr, Mathias, Kügler, Sebastian

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 960 KB

english, 2012

91

Stem Cells and Regenerative Medicine in Lung Biology and Diseases

Lau, Allison N, Goodwin, Meagan, Kim, Carla F, Weiss, Daniel J

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 989 KB

english, 2012

92

Retroviral Integrations in Gene Therapy Trials

Biasco, Luca, Baricordi, Cristina, Aiuti, Alessandro

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 232 KB

english, 2012

93

Targeted Expression of miR-34a Using the T-VISA System Suppresses Breast Cancer Cell Growth and Invasion

Li, Laisheng, Xie, Xinhua, Luo, Jinmei, Liu, Min, Xi, Shaoyan, Guo, Jiaoli, Kong, Yanan, Wu, Minqing, Gao, Jie, Xie, Zeming, Tang, Jun, Wang, Xi, Wei, Weidong, Yang, Mingtian, Hung, Mien-Chie, Xie, Xi

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 697 KB

english, 2012

94

Targeting the Hemoglobin Scavenger receptor CD163 in Macrophages Highly Increases the Anti-inflammatory Potency of Dexamethasone

Graversen, Jonas H, Svendsen, Pia, Dagnæs-Hansen, Frederik, Dal, Jakob, Anton, Gabriele, Etzerodt, Anders, Petersen, Mikkel D, Christensen, Peter A, Møller, Holger J, Moestrup, Søren K

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 523 KB

english, 2012

95

Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular Atrophy

Osman, Erkan Y, Yen, Pei-Fen, Lorson, Christian L

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 313 KB

english, 2012

96

Mesenchymal Stromal Cells Protect Cancer Cells From ROS-induced Apoptosis and Enhance the Warburg Effect by Secreting STC1

Ohkouchi, Shinya, Block, Gregory J, Katsha, Ahmed M, Kanehira, Masahiko, Ebina, Masahito, Kikuchi, Toshiaki, Saijo, Yasuo, Nukiwa, Toshihiro, Prockop, Darwin J

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 279 KB

english, 2012

97

Mesenchymal Stem/Stromal Cells (MSCs): Role as Guardians of Inflammation

Prockop, Darwin J, Youn Oh, Joo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 389 KB

english, 2012

98

Long-term Survival of the Juvenile Lethal Arginase-deficient Mouse With AAV Gene Therapy

Lee, Eun K., Hu, Chuhong, Bhargava, Ragini, Rozengurt, Nora, Stout, David, Grody, Wayne W, Cederbaum, Stephen D, Lipshutz, Gerald S

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.00 MB

english, 2012

99

Normal Collagen and Bone Production by Gene-targeted Human Osteogenesis Imperfecta iPSCs

Deyle, David R, Khan, Iram F, Ren, Gaoying, Wang, Pei-Rong, Kho, Jordan, Schwarze, Ulrike, Russell, David W

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 896 KB

english, 2012

100

Transient Focal Membrane Deformation Induced by Arginine-rich Peptides Leads to Their Direct Penetration into Cells

Hirose, Hisaaki, Takeuchi, Toshihide, Osakada, Hiroko, Pujals, Sílvia, Katayama, Sayaka, Nakase, Ikuhiko, Kobayashi, Shouhei, Haraguchi, Tokuko, Futaki, Shiroh

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 622 KB

english, 2012

101

Inducible IL10+ Suppressor B Cells Inhibit CNS Inflammation and T Helper 17 Polarization

Hsieh, Jeremy, Williams, Patrick, Rafei, Moutih, Birman, Elena, Cuerquis, Jessica, Yuan, Shala, Wu, JianHui, Galipeau, Jacques

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.96 MB

english, 2012

102

Enhancing Muscle Membrane Repair by Gene Delivery of MG53 Ameliorates Muscular Dystrophy and Heart Failure in δ-Sarcoglycan-deficient Hamsters

He, Bo, Tang, Ru-hang, Weisleder, Noah, Xiao, Bin, Yuan, Zhenhua, Cai, Chuanxi, Zhu, Hua, Lin, Peihui, Qiao, Chunping, Li, Jianbin, Mayer, Christina, Li, Juan, Ma, Jianjie, Xiao, Xiao

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 869 KB

english, 2012

103

Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

Keeler, Allison M, Conlon, Thomas, Walter, Glenn, Zeng, Huadong, Shaffer, Scott A, Dungtao, Fu, Erger, Kirsten, Cossette, Travis, Tang, Qiushi, Mueller, Christian, Flotte, Terence R

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 507 KB

english, 2012

104

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

Mingozzi, Federico, Chen, Yifeng, Murphy, Samuel L, Edmonson, Shyrie C, Tai, Alex, Price, Sandra D, Metzger, Mark E, Zhou, Shangzhen, Wright, J Fraser, Donahue, Robert E, Dunbar, Cynthia E, High, Kath

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 231 KB

english, 2012

105

Myoblasts Derived From Normal hESCs and Dystrophic hiPSCs Efficiently Fuse With Existing Muscle Fibers Following Transplantation

Goudenege, Sébastien, Lebel, Carl, Huot, Nicolas B, Dufour, Christine, Fujii, Isao, Gekas, Jean, Rousseau, Joël, Tremblay, Jacques P

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 11.89 MB

english, 2012

106

Co-administration With the Pharmacological Chaperone AT1001 Increases Recombinant Human α-Galactosidase A Tissue Uptake and Improves Substrate Reduction in Fabry Mice

Benjamin, Elfrida R, Khanna, Richie, Schilling, Adriane, Flanagan, John J, Pellegrino, Lee J, Brignol, Nastry, Lun, Yi, Guillen, Darlene, Ranes, Brian E, Frascella, Michelle, Soska, Rebecca, Feng, Jes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 864 KB

english, 2012

107

MyD88 Signaling in B Cells Regulates the Production of Th1-dependent Antibodies to AAV

Sudres, Muriel, Ciré, Séverine, Vasseur, Virginie, Brault, Léa, Da Rocha, Sylvie, Boisgérault, Florence, Le Bec, Christine, Gross, David Alexandre, Blouin, Véronique, Ryffel, Bernard, Galy, Anne

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 387 KB

english, 2012

108

Harnessing a Physiologic Mechanism for siRNA Delivery With Mimetic Lipoprotein Particles

Nakayama, Tomoko, Butler, James S, Sehgal, Alfica, Severgnini, Mariano, Racie, Tim, Sharman, Jennifer, Ding, Feng, Morskaya, Svetlana Shulga, Brodsky, Joshua, Tchangov, Lubomir, Kosovrasti, Verbena, M

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 492 KB

english, 2012

109

Human MSC Suppression Correlates With Cytokine Induction of Indoleamine 2,3-Dioxygenase and Bystander M2 Macrophage Differentiation

François, Moïra, Romieu-Mourez, Raphaëlle, Li, Mengyang, Galipeau, Jacques

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 416 KB

english, 2012

110

Safe, Long-term Hepatic Expression of Anti-HCV shRNA in a Nonhuman Primate Model

Suhy, David A, Kao, Shih-Chu, Mao, Tin, Whiteley, Laurence, Denise, Hubert, Souberbielle, Bernard, Burdick, Andrew D, Hayes, Kyle, Wright, J Fraser, Lavender, Helen, Roelvink, Peter, Kolykhalov, Alexa

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.62 MB

english, 2012

111

The Oncolytic Poxvirus JX-594 Selectively Replicates in and Destroys Cancer Cells Driven by Genetic Pathways Commonly Activated in Cancers

Parato, Kelley A, Breitbach, Caroline J, Le Boeuf, Fabrice, Wang, Jiahu, Storbeck, Chris, Ilkow, Carolina, Diallo, Jean-Simon, Falls, Theresa, Burns, Joseph, Garcia, Vanessa, Kanji, Femina, Evgin, Lau

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 750 KB

english, 2012

112

A Designed Zinc-finger Transcriptional Repressor of Phospholamban Improves Function of the Failing Heart

Zhang, H Steve, Liu, Dingang, Huang, Yan, Schmidt, Stefan, Hickey, Reed, Guschin, Dmitry, Su, Haili, Jovin, Ion S, Kunis, Mike, Hinkley, Sarah, Liang, Yuxin, Hinh, Linda, Spratt, S Kaye, Case, Casey C

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

113

Challenges in Vector and Trial Design Using Retroviral Vectors for Long-Term Gene Correction in Hematopoietic Stem Cell Gene Therapy

Corrigan-Curay, Jacqueline, Cohen-Haguenauer, Odile, O’Reilly, Marina, Ross, Susan R., Fan, Hung, Rosenberg, Naomi, Somia, Nikunj, King, Nancy, Friedmann, Theodore, Dunbar, Cynthia, Aiuti, Alessandro,

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 302 KB

english, 2012

114

Design and Selection of Toca 511 for Clinical Use: Modified Retroviral Replicating Vector With Improved Stability and Gene Expression

Perez, Omar D, Logg, Christopher R, Hiraoka, Kei, Diago, Oscar, Burnett, Ryan, Inagaki, Akihito, Jolson, Dawn, Amundson, Karin, Buckley, Taylor, Lohse, Dan, Lin, Amy, Burrascano, Cindy, Ibanez, Carlos

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.71 MB

english, 2012

115

Antitumor Activity of Cell-Permeable p18INK4c With Enhanced Membrane and Tissue Penetration

Lim, Junghee, Kim, Jungeun, Duong, Tam, Lee, Guewha, Kim, Junghee, Yoon, Jina, Kim, Jaetaek, Kim, Hyuncheol, Ruley, H Earl, El-Rifai, Wael, Jo, Daewoong

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 816 KB

english, 2012

116

Intranasal Delivery of HMGB1 siRNA Confers Target Gene Knockdown and Robust Neuroprotection in the Postischemic Brain

Kim, Il-Doo, Shin, Joo-Hyun, Kim, Seung-Woo, Choi, Sunghyun, Ahn, Junseong, Han, Pyung-Lim, Park, Jong-Sang, Lee, Ja-Kyeong

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.63 MB

english, 2012

117

Nonviral Delivery of Small Interfering RNA Into Pancreas-associated Immune Cells Prevents Autoimmune Diabetes

Leconet, Wilhem, Petit, Pierre, Peraldi-Roux, Sylvie, Bresson, Damien

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 974 KB

english, 2012

118

Rapidly Generated Multivirus-specific Cytotoxic T Lymphocytes for the Prophylaxis and Treatment of Viral Infections

Gerdemann, Ulrike, Keirnan, Jacqueline M, Katari, Usha L, Yanagisawa, Ryu, Christin, Anne S, Huye, Leslie E, Perna, Serena K, Ennamuri, Sravya, Gottschalk, Stephen, Brenner, Malcolm K, Heslop, Helen E

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 469 KB

english, 2012

119

Phase I Trial of “bi-shRNAifurin/GMCSF DNA/Autologous Tumor Cell” Vaccine (FANG) in Advanced Cancer

Senzer, Neil, Barve, Minal, Kuhn, Joseph, Melnyk, Anton, Beitsch, Peter, Lazar, Martin, Lifshitz, Samuel, Magee, Mitchell, Oh, Jonathan, Mill, Susan W, Bedell, Cynthia, Higgs, Candice, Kumar, Padmasin

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 301 KB

english, 2012

120

The AAV Vector Toolkit: Poised at the Clinical Crossroads

Asokan, Aravind, Schaffer, David V, Jude Samulski, R

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 225 KB

english, 2012

121

In Vivo Delivery of Cytoplasmic RNA Virus-derived miRNAs

Langlois, Ryan A, Shapiro, Jillian S, Pham, Alissa M, tenOever, Benjamin R

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 931 KB

english, 2012

122

Lentiviral Vectors Displaying Modified Measles Virus gp Overcome Pre-existing Immunity in In Vivo-like Transduction of Human T and B Cells

Lévy, Camille, Amirache, Fouzia, Costa, Caroline, Frecha, Cecilia, Muller, Claude P, Kweder, Hasan, Buckland, Robin, Cosset, François-Loïc, Verhoeyen, Els

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.38 MB

english, 2012

123

1α,25(OH)2-Vitamin D3 Increases Dysferlin Expression in vitro and in a Human Clinical Trial

Luna, Noemi De, Díaz-Manera, Jordi, Paradas, Carmen, Iturriaga, Cristina, Rojas-García, Ricardo, Araque, Josefa, Genebriera, Mireia, Gich, Ignasi, Illa, Isabel, Gallardo, Eduard

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1013 KB

english, 2012

124

Lentiviral Vector Induced Insertional Haploinsufficiency of Ebf1 Causes Murine Leukemia

Heckl, Dirk, Schwarzer, Adrian, Haemmerle, Reinhard, Steinemann, Doris, Rudolph, Cornelia, Skawran, Britta, Knoess, Sabine, Krause, Johanna, Li, Zhixiong, Schlegelberger, Brigitte, Baum, Christopher,

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 865 KB

english, 2012

125

Alpharetroviral Self-inactivating Vectors: Long-term Transgene Expression in Murine Hematopoietic Cells and Low Genotoxicity

Suerth, Julia D, Maetzig, Tobias, Brugman, Martijn H, Heinz, Niels, Appelt, Jens-Uwe, Kaufmann, Kerstin B, Schmidt, Manfred, Grez, Manuel, Modlich, Ute, Baum, Christopher, Schambach, Axel

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 243 KB

english, 2012

126

Restoration of Vision in the pde6β-deficient Dog, a Large Animal Model of Rod-cone Dystrophy

Petit, Lolita, Lhériteau, Elsa, Weber, Michel, Le Meur, Guylène, Deschamps, Jack-Yves, Provost, Nathalie, Mendes-Madeira, Alexandra, Libeau, Lyse, Guihal, Caroline, Colle, Marie-Anne, Moullier, Philip

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 3.12 MB

english, 2012

127

Differing Approaches to Experimental Therapeutics: Are We a World Apart?

Cripe, Timothy P

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 337 KB

english, 2012

128

PCSK9 LNA Antisense Oligonucleotides Induce Sustained Reduction of LDL Cholesterol in Nonhuman Primates

Lindholm, Marie W, Elmén, Joacim, Fisker, Niels, Hansen, Henrik F, Persson, Robert, Møller, Marianne R, Rosenbohm, Christoph, Ørum, Henrik, Straarup, Ellen M, Koch, Troels

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 234 KB

english, 2012

129

AAV Transduction of Dopamine Neurons With Constitutively Active Rheb Protects From Neurodegeneration and Mediates Axon Regrowth

Kim, Sang Ryong, Kareva, Tatyana, Yarygina, Olga, Kholodilov, Nikolai, Burke, Robert E

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.20 MB

english, 2012

130

Cancer Immunotherapy Using a Membrane-bound Interleukin-12 With B7-1 Transmembrane and Cytoplasmic Domains

Pan, Wen-Yu, Lo, Chia-Hui, Chen, Chun-Chi, Wu, Ping-Yi, Roffler, Steve R, Shyue, Song-Kun, Tao, Mi-Hua

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.07 MB

english, 2012

131

Short-interfering RNAs Induce Retinal Degeneration via TLR3 and IRF3

Kleinman, Mark E, Kaneko, Hiroki, Cho, Won Gil, Dridi, Sami, Fowler, Benjamin J, Blandford, Alexander D, Albuquerque, Romulo JC, Hirano, Yoshio, Terasaki, Hiroko, Kondo, Mineo, Fujita, Takashi, Ambati

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1021 KB

english, 2012

132

Adenovirus-mediated E2-EPF UCP Gene Transfer Prevents Autoamputation in a Mouse Model of Hindlimb Ischemia

Lim, Jung Hwa, Shin, Hyo Jung, Park, Kyeong-Su, Lee, Chan Hee, Jung, Cho-Rok, Im, Dong-Soo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.33 MB

english, 2012

133

Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease

Rafi, Mohammad A, Rao, Han Zhi, Luzi, Paola, Curtis, Mark T, Wenger, David A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 7.15 MB

english, 2012

134

Late Abstracts: Presented at the American Society of Gene & Cell Therapy’s 15th Annual Meeting, May 16–19, 2012, Philadelphia, Pennsylvania

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 366 KB

english, 2012

135

Ex Vivo Adenoviral Vector Gene Delivery Results in Decreased Vector-associated Inflammation Pre- and Post–lung Transplantation in the Pig

Yeung, Jonathan C, Wagnetz, Dirk, Cypel, Marcelo, Rubacha, Matthew, Koike, Terumoto, Chun, Yi-Min, Hu, Jim, Waddell, Thomas K, Hwang, David M, Liu, Mingyao, Keshavjee, Shaf

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 785 KB

english, 2012

136

Nonmyeloablative Conditioning Regimen to Increase Engraftment of Gene-modified Hematopoietic Stem Cells in Young Rhesus Monkeys

Tarantal, Alice F, Giannoni, Francesca, I Lee, C Chang, Wherley, Jennifer, Sumiyoshi, Teiko, Martinez, Michele, Kahl, Christoph A, Elashoff, David, Louie, Stan G, Kohn, Donald B

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.94 MB

english, 2012

137

Antitumor Efficacy of 34.5ENVE: A Transcriptionally Retargeted and “Vstat120”-expressing Oncolytic Virus

Yoo, Ji Young, Haseley, Amy, Bratasz, Anna, Chiocca, E Antonio, Zhang, Jianying, Powell, Kimerly, Kaur, Balveen

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.37 MB

english, 2012

138

Delivery of siRNA to the Mouse Lung via a Functionalized Lipopolyamine

Polach, Kevin J, Matar, Majed, Rice, Jennifer, Slobodkin, Gregory, Sparks, Jeff, Congo, Richard, Rea-Ramsey, Angela, McClure, Diane, Brunhoeber, Elaine, Krampert, Monika, Schuster, Andrea, Jahn-Hofman

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 489 KB

english, 2012

139

Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice

Qiu, Kai, Falk, Darin J, Reier, Paul J, Byrne, Barry J, Fuller, David D

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 396 KB

english, 2012

140

Striatal Pleiotrophin Overexpression Provides Functional and Morphological Neuroprotection in the 6-Hydroxydopamine Model

Gombash, Sara E, Lipton, Jack W, Collier, Timothy J, Madhavan, Lalitha, Steece-Collier, Kathy, Cole-Strauss, Allyson, Terpstra, Brian T, Spieles-Engemann, Anne L, Daley, Brian F, Wohlgenant, Susan L,

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 681 KB

english, 2012

141

Liver Production of Sulfamidase Reverses Peripheral and Ameliorates CNS Pathology in Mucopolysaccharidosis IIIA Mice

Ruzo, Albert, Garcia, Miquel, Ribera, Albert, Villacampa, Pilar, Haurigot, Virginia, Marcó, Sara, Ayuso, Eduard, Anguela, Xavier M, Roca, Carles, Agudo, Judith, Ramos, David, Ruberte, Jesús, Bosch, Fa

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.68 MB

english, 2012

142

An Artificial PAP Gene Breaks Self-tolerance and Promotes Tumor Regression in the TRAMP Model for Prostate Carcinoma

Spies, Elmar, Reichardt, Wilfried, Alvarez, Gerardo, Groettrup, Marcus, öhlschläger, Peter

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 355 KB

english, 2012

143

Redirected Antitumor Activity of Primary Human Lymphocytes Transduced With a Fully Human Anti-mesothelin Chimeric Receptor

Lanitis, Evripidis, Poussin, Mathilde, Hagemann, Ian S, Coukos, George, Sandaltzopoulos, Raphael, Scholler, Nathalie, Powell, Daniel J

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 507 KB

english, 2012

144

NF-κB Negatively Impacts the Myogenic Potential of Muscle-derived Stem Cells

Lu, Aiping, Proto, Jonathan D, Guo, Lulin, Tang, Ying, Lavasani, Mitra, Tilstra, Jeremy S, Niedernhofer, Laura J, Wang, Bing, Guttridge, Denis C, Robbins, Paul D, Huard, Johnny

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 639 KB

english, 2012

145

Concomitant Intravenous Nitroglycerin With Intracoronary Delivery of AAV1.SERCA2a Enhances Gene Transfer in Porcine Hearts

Karakikes, Ioannis, Hadri, Lahouaria, Rapti, Kleopatra, Ladage, Dennis, Ishikawa, Kiyotake, Tilemann, Lisa, Yi, Geng-Hua, Morel, Charlotte, Gwathmey, Judith K, Zsebo, Krisztina, Weber, Thomas, Kawase,

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 272 KB

english, 2012

146

Live and Let Die: A New Suicide Gene Therapy Moves to the Clinic

Morgan, Richard A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 272 KB

english, 2012

147

Multifunctional Theranostic Nanoparticles for Brain Tumors

Wang, Yuhua, Huang, Leaf

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 248 KB

english, 2012

148

Antisense Oligonucleotides Shed New Light on the Pathogenesis and Treatment of Spinal Muscular Atrophy

Yee, Jiing-Kuan, Lin, Ren-Jang

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

149

Combined mTOR Inhibition and OX40 Agonism Enhances CD8+ T Cell Memory and Protective Immunity Produced by Recombinant Adenovirus Vaccines

Bassett, Jennifer D, Swift, Stephanie L, VanSeggelen, Heather, Hammill, Joanne A, McGray, AJ Robert, Evelegh, Carole, Wan, Yonghong, Bramson, Jonathan L

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 701 KB

english, 2012

150

Combined Tbet and IL12 Gene Therapy Elicits and Recruits Superior Antitumor Immunity In Vivo

Qu, Yanyan, Chen, Lu, Lowe, Devin B, Storkus, Walter J, Taylor, Jennifer L

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 476 KB

english, 2012

151

Soluble Vascular Endothelial Growth Factor Decoy Receptor FP3 Exerts Potent Antiangiogenic Effects

Yu, De-Chao, Lee, Jung-Sun, Yoo, Ji Young, Shin, Hyewon, Deng, Hongxin, Wei, Yuquan, Yun, Chae-Ok

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 501 KB

english, 2012

152

Directing Integrin-linked Endocytosis of Recombinant AAV Enhances Productive FAK-dependent Transduction

Kaminsky, Paul M, Keiser, Nicholas W, Yan, Ziying, Lei-Butters, Diana CM, Engelhardt, John F

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.12 MB

english, 2012

153

Signs of Progress in Gene Therapy for Muscular Dystrophy Also Warrant Caution

Stedman, Hansell H, Byrne, Barry J

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 233 KB

english, 2012

154

Design of a Single AAV Vector for Coexpression of TH and GCH1 to Establish Continuous DOPA Synthesis in a Rat Model of Parkinson's Disease

Cederfjäll, Erik, Sahin, Gurdal, Kirik, Deniz, Björklund, Tomas

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 350 KB

english, 2012

155

HIV Develops Indirect Cross-resistance to Combinatorial RNAi Targeting Two Distinct and Spatially Distant Sites

Shah, Priya S, Pham, Nhung P, Schaffer, David V

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 631 KB

english, 2012

156

A Water-Soluble Coelenterazine for Sensitive In Vivo Imaging of Coelenterate Luciferases

Morse, Danielle, Tannous, Bakhos A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 486 KB

english, 2012

157

Do Cell-Penetrating Peptides Actually “Penetrate” Cellular Membranes?

Palm-Apergi, Caroline, Lönn, Peter, Dowdy, Steven F

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 908 KB

english, 2012

158

Seeing Eye to Eye With Gene Therapy

Herzog, Roland W

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 449 KB

english, 2012

159

Integration Frequency and Intermolecular Recombination of rAAV Vectors in Non-human Primate Skeletal Muscle and Liver

Nowrouzi, Ali, Penaud-Budloo, Magalie, Kaeppel, Christine, Appelt, Uwe, Le Guiner, Caroline, Moullier, Philippe, Kalle, Christof von, Snyder, Richard O, Schmidt, Manfred

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 387 KB

english, 2012

160

Lens Epithelium-derived Growth Factor/p75 Qualifies as a Target for HIV Gene Therapy in the NSG Mouse Model

Vets, Sofie, Kimpel, Janine, Volk, Andreas, De Rijck, Jan, Schrijvers, Rik, Verbinnen, Bert, Maes, Wim, Von Laer, Dorothee, Debyser, Zeger, Gijsbers, Rik

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 526 KB

english, 2012

161

A Small-molecule-controlled System for Efficient Pseudotyping of Prototype Foamy Virus Vectors

Ho, Yu-Ping, Schnabel, Viktor, Swiersy, Anka, Stirnnagel, Kristin, Lindemann, Dirk

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 506 KB

english, 2012

162

Increased Erythropoiesis in Mice Injected With Submicrogram Quantities of Pseudouridine-containing mRNA Encoding Erythropoietin

Karikó, Katalin, Muramatsu, Hiromi, Keller, Jason M, Weissman, Drew

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 171 KB

english, 2012

163

Enforced IL-10 Expression Confers Type 1 Regulatory T Cell (Tr1) Phenotype and Function to Human CD4+ T Cells

Andolfi, Grazia, Fousteri, Georgia, Rossetti, Maura, Magnani, Chiara F, Jofra, Tatiana, Locafaro, Grazia, Bondanza, Attilio, Gregori, Silvia, Roncarolo, Maria-Grazia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 2.30 MB

english, 2012

164

Local In Vivo GSK3β Knockdown Promotes Pancreatic β Cell and Acinar Cell Regeneration in 90% Pancreatectomized Rat

Figeac, Florence, Ilias, Anissa, Bailbe, Danielle, Portha, Bernard, Movassat, Jamileh

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.05 MB

english, 2012

165

Selectins for Cardiosphere Culture: The “E’s” Have It!

Davis, Darryl R, Stewart, Duncan J

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 208 KB

english, 2012

166

Converting Scar to Muscle in the Injured Heart

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.68 MB

english, 2012

167

Administration of Anti-CD3 Antibodies Modulates the Immune Response to an Infusion of α-glucosidase in Mice

Ohashi, Toya, Iizuka, Sayoko, Shimada, Yohta, Higuchi, Takashi, Eto, Yoshikatsu, Ida, Hiroyuki, Kobayashi, Hiroshi

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 482 KB

english, 2012

168

Phase II Trial of Intravenous Administration of Reolysin® (Reovirus Serotype-3-dearing Strain) in Patients with Metastatic Melanoma

Galanis, Evanthia, Markovic, Svetomir N, Suman, Vera J, Nuovo, Gerard J, Vile, Richard G, Kottke, Timothy J, Nevala, Wendy K, Thompson, Michael A, Lewis, Jean E, Rumilla, Kandelaria M, Roulstone, Vict

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.79 MB

english, 2012

169

In Vivo Selection of Transplanted Hepatocytes by Pharmacological Inhibition of Fumarylacetoacetate Hydrolase in Wild-type Mice

Paulk, Nicole K, Wursthorn, Karsten, Haft, Annelise, Pelz, Carl, Clarke, Gregory, Newell, Amy H, Olson, Susan B, Harding, Cary O, Finegold, Milton J, Bateman, Raymond L, Witte, John F, McClard, Ronald

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.72 MB

english, 2012

170

Hematopoietic Stem Cell and Gene Therapy Corrects Primary Neuropathology and Behavior in Mucopolysaccharidosis IIIA Mice

Langford-Smith, Alexander, Wilkinson, Fiona L, Langford-Smith, Kia J, Holley, Rebecca J, Sergijenko, Ana, Howe, Steven J, Bennett, William R, Jones, Simon A, Wraith, JE, Merry, Catherine LR, Wynn, Rob

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 737 KB

english, 2012

171

LNA-based Oligonucleotide Electrotransfer for miRNA Inhibition

Chabot, Sophie, Orio, Julie, Castanier, Romain, Bellard, Elisabeth, Nielsen, Søren J, Golzio, Muriel, Teissié, Justin

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 573 KB

english, 2012

172

Sialic Acid Deposition Impairs the Utility of AAV9, but Not Peptide-modified AAVs for Brain Gene Therapy in a Mouse Model of Lysosomal Storage Disease

Chen, Yong Hong, Claflin, Kristin, Geoghegan, James C, Davidson, Beverly L

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 471 KB

english, 2012

173

Cell Fate Control Gene Therapy Based on Engineered Variants of Human Deoxycytidine Kinase

Neschadim, Anton, Wang, James CM, Sato, Takeya, Fowler, Daniel H, Lavie, Arnon, Medin, Jeffrey A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 694 KB

english, 2012

174

High-efficiency Transduction of Rhesus Hematopoietic Repopulating Cells by a Modified HIV1-based Lentiviral Vector

Uchida, Naoya, Hargrove, Phillip W., Lap, Coen J., Evans, Molly E., Phang, Oswald, Bonifacino, Aylin C., Krouse, Allen E., Metzger, Mark E., Nguyen, Anh-Dao, Hsieh, Matthew M., Wolfsberg, Tyra G., Don

Journal:

Molecular Therapy

Year:

2012

File:

PDF, 1.16 MB

2012

175

Long-term Engraftment of Multipotent Mesenchymal Stromal Cells That Differentiate to Form Myogenic Cells in Dogs With Duchenne Muscular Dystrophy

Nitahara-Kasahara, Yuko, Hayashita-Kinoh, Hiromi, Ohshima-Hosoyama, Sachiko, Okada, Hironori, Wada-Maeda, Michiko, Nakamura, Akinori, Okada, Takashi, Takeda, Shin'ichi

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 866 KB

english, 2012

176

Optimization of hCFTR Lung Expression in Mice Using DNA Nanoparticles

Padegimas, Linas, Kowalczyk, Tomasz H, Adams, Sam, Gedeon, Chris R, Oette, Sharon M, Dines, Karla, Hyatt, Susannah L, Sesenoglu-Laird, Ozge, Tyr, Olena, Moen, Robert C, Cooper, Mark J

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 273 KB

english, 2012

177

Rapidly Regulating Platelet Activity In Vivo With an Antidote Controlled Platelet Inhibitor

Nimjee, Shahid M, Lohrmann, Jens D, Wang, Haichen, Snyder, David J, Cummings, Thomas J, Becker, Richard C, Oney, Sabah, Sullenger, Bruce A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 227 KB

english, 2012

178

Preclinical Dose-Finding Study With a Liver-Tropic, Recombinant AAV-2/8 Vector in the Mouse Model of Galactosialidosis

Hu, Huimin, Gomero, Elida, Bonten, Erik, Gray, John T, Allay, Jim, Wu, Yanan, Wu, Jianrong, Calabrese, Christopher, Nienhuis, Arthur, d'Azzo, Alessandra

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.15 MB

english, 2012

179

Human Skeletal Muscle Cells With a Slow Adhesion Rate After Isolation and an Enhanced Stress Resistance Improve Function of Ischemic Hearts

Okada, Masaho, Payne, Thomas R, Drowley, Lauren, Jankowski, Ron J, Momoi, Nobuo, Beckman, Sarah, Chen, William CW, Keller, Bradley B, Tobita, Kimimasa, Huard, Johnny

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 427 KB

english, 2012

180

Is Serum IL-17A a Useful Systemic Biomarker in Patients With Langerhans Cell Histiocytosis?

Makras, Polyzois, Polyzos, Stergios A, Anastasilakis, Athanasios D, Terpos, Evangelos, Papatheodorou, Athanasios, Kaltsas, Gregory A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 91 KB

english, 2012

181

Self-complementary AAVs Induce More Potent Transgene Product-specific Immune Responses Compared to a Single-stranded Genome

Wu, TeLang, Töpfer, Katrin, Lin, Shih-Wen, Li, Hua, Bian, Ang, Zhou, Xiang Y, High, Katherine A, Ertl, Hildegund CJ

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 376 KB

english, 2012

182

Long-term Persistence of CD4+ but Rapid Disappearance of CD8+ T Cells Expressing an MHC Class I-restricted TCR of Nanomolar Affinity

Engels, Boris, Chervin, Adam S, Sant, Andrea J, Kranz, David M, Schreiber, Hans

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 453 KB

english, 2012

183

Suprachoroidal Electrotransfer: A Nonviral Gene Delivery Method to Transfect the Choroid and the Retina Without Detaching the Retina

Touchard, Elodie, Berdugo, Marianne, Bigey, Pascal, El Sanharawi, Mohamed, Savoldelli, Michèle, Naud, Marie-Christine, Jeanny, Jean-Claude, Behar-Cohen, Francine

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.24 MB

english, 2012

184

BioKnife, a uPA Activity-dependent Oncolytic Sendai Virus, Eliminates Pleural Spread of Malignant Mesothelioma via Simultaneous Stimulation of uPA Expression

Morodomi, Yosuke, Yano, Tokujiro, Kinoh, Hiroaki, Harada, Yui, Saito, Satoru, Kyuragi, Ryoichi, Yoshida, Kumi, Onimaru, Mitsuho, Shoji, Fumihiro, Yoshida, Tsukihisa, Ito, Kensaku, Shikada, Yasunori, M

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 541 KB

english, 2012

185

Gene Transfer to the CNS Is Efficacious in Immune-primed Mice Harboring Physiologically Relevant Titers of Anti-AAV Antibodies

Treleaven, Christopher M, Tamsett, Thomas J, Bu, Jie, Fidler, Jonathan A, Sardi, S Pablo, Hurlbut, Gregory D, Woodworth, Lisa A, Cheng, Seng H, Passini, Marco A, Shihabuddin, Lamya S, Dodge, James C

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 3.81 MB

english, 2012

186

Ad3-hTERT-E1A, a Fully Serotype 3 Oncolytic Adenovirus, in Patients With Chemotherapy Refractory Cancer

Hemminki, Otto, Diaconu, Iulia, Cerullo, Vincenzo, Pesonen, Saila K, Kanerva, Anna, Joensuu, Timo, Kairemo, Kalevi, Laasonen, Leena, Partanen, Kaarina, Kangasniemi, Lotta, Lieber, Andre, Pesonen, Sari

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 661 KB

english, 2012

187

T-Cell Therapy for Viral Infections Following Transplantation: Why Stop at Three Viruses?

Barrett, John A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 213 KB

english, 2012

188

Assessing Journal Influence: Impacted Wisdom

Frederickson, Robert M, Brenner, Malcolm K

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 194 KB

english, 2012

189

Thymic Gene Transfer of Myelin Oligodendrocyte Glycoprotein Ameliorates the Onset but Not the Progression of Autoimmune Demyelination

Siatskas, Christopher, Seach, Natalie, Sun, Guizhi, Emerson-Webber, Ashley, Silvain, Aude, Toh, Ban-Hock, Alderuccio, Frank, Bäckström, B Thomas, Boyd, Richard L, Bernard, Claude C

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 837 KB

english, 2012

190

AAV Vectors Containing rDNA Homology Display Increased Chromosomal Integration and Transgene Persistence

Wang, Zhongya, Lisowski, Leszek, Finegold, Milton J, Nakai, Hiroyuki, Kay, Mark A, Grompe, Markus

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 689 KB

english, 2012

191

Thymidine Kinase Suicide Gene-mediated Ganciclovir Ablation of Autologous Gene-modified Rhesus Hematopoiesis

Barese, Cecilia N, Krouse, Allen E, Metzger, Mark E, King, Connor A, Traversari, Catia, Marini, Frank C, Donahue, Robert E, Dunbar, Cynthia E

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 809 KB

english, 2012

192

All for One, One for All: New Combinatorial RNAi Therapies Combat Hepatitis C Virus Evolution

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.31 MB

english, 2012

193

Taking a Break From Huntingtin

Davidson, Beverly L

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

194

Conditional Negative Selection of Gene-Modified Hematopoietic Stem Cells

Baum, Christopher

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 249 KB

english, 2012

195

Buffering Capacity and Size of siRNA Polyplexes Influence Cytokine Levels

Leng, Qixin, Chou, Szu-Ting, Scaria, Puthupparampil V, Woodle, Martin C, Mixson, A James

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 591 KB

english, 2012

196

A New Vision of Mesenchymal Stromal Cells

Hurwitz, Richard L

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 248 KB

english, 2012

197

European Union Support of Gene Therapy Research: A Success Story

Gancberg, David, Kessler, Charles, Hoeveler, Arnd, Draghia-Akli, Ruxandra

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 478 KB

english, 2012

198

UU/UA Dinucleotide Frequency Reduction in Coding Regions Results in Increased mRNA Stability and Protein Expression

Al-Saif, Maher, Khabar, Khalid SA

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 378 KB

english, 2012

199

Aire Controls Mesenchymal Stem Cell-mediated Suppression in Chronic Colitis

Parekkadan, Biju, Fletcher, Anne L, Li, Matthew, Tjota, Melissa Y, Bellemare-Pelletier, Angelique, Milwid, Jack M, Lee, Je-Wook, Yarmush, Martin L, Turley, Shannon J

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 531 KB

english, 2012

200

Hematopoietic Stem Cell Mobilization for Gene Therapy of Adult Patients With Severe β-Thalassemia: Results of Clinical Trials Using G-CSF or Plerixafor in Splenectomized and Nonsplenectomized Subjects

Yannaki, Evangelia, Papayannopoulou, Thalia, Jonlin, Erica, Zervou, Fani, Karponi, Garyfalia, Xagorari, Angeliki, Becker, Pamela, Psatha, Nikoleta, Batsis, Ioannis, Kaloyannidis, Panayotis, Tahynopoul

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 228 KB

english, 2012

201

Construction of an Oncolytic Herpes Simplex Virus That Precisely Targets Hepatocellular Carcinoma Cells

Fu, Xinping, Rivera, Armando, Tao, Lihua, De Geest, Bart, Zhang, Xiaoliu

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

202

ORFV: A Novel Oncolytic and Immune Stimulating Parapoxvirus Therapeutic

Rintoul, Julia L, Lemay, Chantal G, Tai, Lee-Hwa, Stanford, Marianne M, Falls, Theresa J, de Souza, Christiano T, Bridle, Byram W, Daneshmand, Manijeh, Ohashi, Pamela S, Wan, Yonghong, Lichty, Brian D

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 496 KB

english, 2012

203

Balloon Catheter Delivery of Helper-dependent Adenoviral Vector Results in Sustained, Therapeutic hFIX Expression in Rhesus Macaques

Brunetti-Pierri, Nicola, Liou, Aimee, Patel, Priti, Palmer, Donna, Grove, Nathan, Finegold, Milton, Piccolo, Pasquale, Donnachie, Elizabeth, Rice, Karen, Beaudet, Arthur, Mullins, Charles, Ng, Philip

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 483 KB

english, 2012

204

The State of Gene Therapies: The FDA Perspective

Takefman, Daniel, Bryan, Wilson

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 190 KB

english, 2012

205

A Cell-penetrating Helical Polymer For siRNA Delivery to Mammalian Cells

Gabrielson, Nathan P, Lu, Hua, Yin, Lichen, Kim, Kyung Hoon, Cheng, Jianjun

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 520 KB

english, 2012

206

High-efficiency Transduction of Rhesus Hematopoietic Repopulating Cells by a Modified HIV1-based Lentiviral Vector

Uchida, Naoya, Hargrove, Phillip W., Lap, Coen J., Evans, Molly E., Phang, Oswald, Bonifacino, Aylin C., Krouse, Allen E., Metzger, Mark E., Nguyen, Anh-Dao, Hsieh, Matthew M., Wolfsberg, Tyra G., Don

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.16 MB

english, 2012

207

Functionally Enhanced siRNA Targeting TNFα Attenuates DSS-induced Colitis and TLR-mediated Immunostimulation in Mice

Ocampo, Sandra M, Romero, Carolina, Aviñó, Anna, Burgueño, Joan, Gassull, Miguel A, Bermúdez, Jordi, Eritja, Ramon, Fernandez, Ester, Perales, Jose C

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 506 KB

english, 2012

208

Modification of the Early Gene Enhancer-promoter Improves the Oncolytic Potency of Adenovirus 11

Wong, Han Hsi, Jiang, Guozhong, Gangeswaran, Rathi, Wang, Pengju, Wang, Jiwei, Yuan, Ming, Wang, Hexiao, Bhakta, Vipul, Müller, Heike, Lemoine, Nicholas R, Wang, Yaohe

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.69 MB

english, 2012

209

Targeted Gene Modification of Hematopoietic Progenitor Cells in Mice Following Systemic Administration of a PNA-peptide Conjugate

Rogers, Faye A, Lin, Sharon S, Hegan, Denise C, Krause, Diane S, Glazer, Peter M

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 746 KB

english, 2012

210

Protein-anchoring Strategy for Delivering Acetylcholinesterase to the Neuromuscular Junction

Ito, Mikako, Suzuki, Yumi, Okada, Takashi, Fukudome, Takayasu, Yoshimura, Toshiro, Masuda, Akio, Takeda, Shin'ichi, Krejci, Eric, Ohno, Kinji

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 448 KB

english, 2012

211

AAV Vectors for the Nucleolus

Russell, David W

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 399 KB

english, 2012

212

ACE2 and Ang-(1-7) Confer Protection Against Development of Diabetic Retinopathy

Verma, Amrisha, Shan, Zhiying, Lei, Bo, Yuan, Lihui, Liu, Xuan, Nakagawa, Takahiko, Grant, Maria B, Lewin, Alfred S, Hauswirth, William W, Raizada, Mohan K, Li, Qiuhong

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.22 MB

english, 2012

213

Personalized Medicine: Words That Mean Just What You Choose?

Brenner, Malcolm K

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 187 KB

english, 2012

214

The Challenge of Using Gene- or Cell-Based Therapies to Treat Lung Disease

Crystal, Ronald G

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 792 KB

english, 2012

215

Verapamil Results in Increased Blood Levels of Oncolytic Adenovirus in Treatment of Patients With Advanced Cancer

Koski, Anniina, Raki, Mari, Nokisalmi, Petri, Liikanen, Ilkka, Kangasniemi, Lotta, Joensuu, Timo, Kanerva, Anna, Pesonen, Sari, Alemany, Ramon, Hemminki, Akseli

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 219 KB

english, 2012

216

Chemoprotection in Brain Tumor Patients: Another Success for Stem Cell Gene Therapy

Sorrentino, Brian P

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 337 KB

english, 2012

217

Loss of miR-29 in Myoblasts Contributes to Dystrophic Muscle Pathogenesis

Wang, Lijun, Zhou, Liang, Jiang, Peiyong, Lu, Leina, Chen, Xiaona, Lan, Huiyao, Guttridge, Denis C, Sun, Hao, Wang, Huating

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 569 KB

english, 2012

218

PD-1/PD-L1 Blockade Can Enhance HIV-1 Gag-specific T Cell Immunity Elicited by Dendritic Cell-Directed Lentiviral Vaccines

Dai, Bingbing, Xiao, Liang, Bryson, Paul D, Fang, Jinxu, Wang, Pin

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.31 MB

english, 2012

219

Slowing Down Differentiation of Engrafted Human Myoblasts Into Immunodeficient Mice Correlates With Increased Proliferation and Migration

Riederer, Ingo, Negroni, Elisa, Bencze, Maximilien, Wolff, Annie, Aamiri, Ahmed, Di Santo, James P, Silva-Barbosa, Suse D., Butler-Browne, Gillian, Savino, Wilson, Mouly, Vincent

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.04 MB

english, 2012

220

Distinct Strategies Are Required to Suppress Antigen-specific Responses to Genetically Modified Keratinocytes and Fibroblasts

Ghazizadeh, Soosan, Huang, Li T, Zhang, Weibing

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 437 KB

english, 2012

221

Introducing a New ASGCT Forum for Publication of Methodology and Clinical Development in Gene, Cell, and Oligonucleotide Therapies

Frederickson, Robert M, Brenner, Malcolm K

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 428 KB

english, 2012

222

It’s Time to End RAC Review of Gene Therapy Protocols

McGarrity, Gerard J

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

223

Gene Therapy by Targeted Adenovirus-mediated Knockdown of Pulmonary Endothelial Tph1 Attenuates Hypoxia-induced Pulmonary Hypertension

Morecroft, Ian, White, Katie, Caruso, Paola, Nilsen, Margaret, Loughlin, Lynn, Alba, Raul, Reynolds, Paul N, Danilov, Sergei M, Baker, Andrew H, MacLean, Margaret R

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 610 KB

english, 2012

224

Neonatal Gene Therapy With a Gamma Retroviral Vector in Mucopolysaccharidosis VI Cats

Ponder, Katherine P, O'Malley, Thomas M, Wang, Ping, O'Donnell, Patricia A, Traas, Anne M, Knox, Van W, Aguirre, Gustavo A, Ellinwood, N Matthew, Metcalf, Jason A, Wang, Bin, Parkinson-Lawrence, Emma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 710 KB

english, 2012

225

Progress in the Development of Hepatitis C Virus Vaccines

Ertl, Hildegund CJ

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 836 KB

english, 2012

226

Cell-Penetrating RNAs: New Keys to the Castle

Burke, Donald H

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 315 KB

english, 2012

227

Mathematical Modeling in Immunotherapy of Cancer: Personalizing Clinical Trials

Agur, Zvia, Vuk-Pavlović, Stanimir

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 213 KB

english, 2012

228

Gene Therapy for Deaf Mice Goes Viral

Holt, Jeffrey R, Vandenberghe, Luk H

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 421 KB

english, 2012

229

Converting Pathological Cells to Therapeutic Ones: An Odyssey Through Pluripotency

Vilquin, Jean-Thomas

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 262 KB

english, 2012

230

mRNA Leapfrogs DNA to Show Promise for Therapeutic Gene Transfer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 897 KB

english, 2012

231

Genetic Targeting of the Active Transcription Factor XBP1s to Dendritic Cells Potentiates Vaccine-induced Prophylactic and Therapeutic Antitumor Immunity

Tian, Shenghe, Liu, Zuqiang, Donahue, Cara, Falo, Louis D, You, Zhaoyang

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 408 KB

english, 2012

232

2A Peptide-based, Lentivirus-mediated Anti-death Receptor 5 Chimeric Antibody Expression Prevents Tumor Growth in Nude Mice

Li, Meng, Wu, Yaomin, Qiu, Yuhe, Yao, Zhenyu, Liu, Shilian, Liu, Yanxin, Shi, Juan, Zheng, Dexian

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 351 KB

english, 2012

233

Ribosomal DNA Integrating rAAV-rDNA Vectors Allow for Stable Transgene Expression

Lisowski, Leszek, Lau, Ashley, Wang, Zhongya, Zhang, Yue, Zhang, Feijie, Grompe, Markus, Kay, Mark A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 705 KB

english, 2012

234

Human Tumor Xenografts: The Good, the Bad, and the Ugly

Morgan, Richard A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

235

Neutralizing Antibodies Against AAV Serotypes 1, 2, 6, and 9 in Sera of Commonly Used Animal Models

Rapti, Kleopatra, Louis-Jeune, Vedell, Kohlbrenner, Erik, Ishikawa, Kiyotake, Ladage, Dennis, Zolotukhin, Sergei, Hajjar, Roger J, Weber, Thomas

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 297 KB

english, 2012

236

Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles

Mueller, Christian, Tang, Qiushi, Gruntman, Alisha, Blomenkamp, Keith, Teckman, Jeffery, Song, Lina, Zamore, Phillip D, Flotte, Terence R

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.02 MB

english, 2012

237

Cell Membrane–associated Heparan Sulfate Is a Receptor for Prototype Foamy Virus in Human, Monkey, and Rodent Cells

Nasimuzzaman, Md, Persons, Derek A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 392 KB

english, 2012

238

Safety and Feasibility of High-pressure Transvenous Limb Perfusion With 0.9% Saline in Human Muscular Dystrophy

Fan, Zheng, Kocis, Keith, Valley, Robert, Howard, James F, Chopra, Manisha, An, Hongyu, Lin, Weili, Muenzer, Joseph, Powers, William

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 415 KB

english, 2012

239

Bevacizumab With Angiostatin-armed oHSV Increases Antiangiogenesis and Decreases Bevacizumab-induced Invasion in U87 Glioma

Zhang, Wei, Fulci, Giulia, Buhrman, Jason S, Stemmer-Rachamimov, Anat O, Chen, John W, Wojtkiewicz, Gregory R, Weissleder, Ralph, Rabkin, Samuel D, Martuza, Robert L

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.23 MB

english, 2012

240

Safety and Tolerability of the Easy Vax™ Clinical Epidermal Electroporation System in Healthy Adults

El-Kamary, Samer S, Billington, Melissa, Deitz, Stephen, Colby, Elaina, Rhinehart, Howard, Wu, Yukun, Blackwelder, William, Edelman, Robert, Lee, Albert, King, Alan

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

241

Is the Induction of Tumor Cell Senescence the Key to a Good Irradiated Tumor Vaccine?

Brown, Michael P

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 820 KB

english, 2012

242

Novel Therapies, High-Risk Pediatric Research, and the Prospect of Benefit: Learning from the Ethical Disagreements

de Melo-Martín, Inmaculada, Sondhi, Dolan, Crystal, Ronald G

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 281 KB

english, 2012

243

Quantitative 3D Tracing of Gene-delivery Viral Vectors in Human Cells and Animal Tissues

Xiao, Ping-Jie, Li, Chengwen, Neumann, Aaron, Samulski, R Jude

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 752 KB

english, 2012

244

Foamy Virus Vector-mediated Gene Correction of a Mouse Model of Wiskott–Aldrich Syndrome

Uchiyama, Toru, Adriani, Marsilio, Jagadeesh, G Jayashree, Paine, Adam, Candotti, Fabio

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 473 KB

english, 2012

245

Gene Transfer Corrects Acute GM2 Gangliosidosis—Potential Therapeutic Contribution of Perivascular Enzyme Flow

Cachón-González, M Begoña, Wang, Susan Z, McNair, Rosamund, Bradley, Josephine, Lunn, David, Ziegler, Robin, Cheng, Seng H, Cox, Timothy M

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.61 MB

english, 2012

246

CD166pos Subpopulation From Differentiated Human ES and iPS Cells Support Repair of Acute Lung Injury

Soh, Boon Seng, Zheng, Dahai, Li Yeo, Julie Su, Yang, Henry He, Ng, Shi Yan, Wong, Lan Hiong, Zhang, Wencai, Li, Pin, Nichane, Massimo, Asmat, Atasha, Wong, Poo Sing, Wong, Peng Cheang, Su, Lin Lin, M

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.97 MB

english, 2012

247

Pushing the Reset Button: Chemical-Induced Conversion of Amniotic Fluid Stem Cells Into a Pluripotent State

Diecke, Sebastian, Wu, Joseph C

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 407 KB

english, 2012

248

Escaping the Valley of Death

Frederickson, Robert M

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 304 KB

english, 2012

249

Efficient and Accurate Homologous Recombination in hESCs and hiPSCs Using Helper-dependent Adenoviral Vectors

Aizawa, Emi, Hirabayashi, Yuka, Iwanaga, Yuzuru, Suzuki, Keiichiro, Sakurai, Kenji, Shimoji, Miho, Aiba, Kazuhiro, Wada, Tamaki, Tooi, Norie, Kawase, Eihachiro, Suemori, Hirofumi, Nakatsuji, Norio, Mi

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 578 KB

english, 2012

250

Myocardial Repair and Regeneration: Bone Marrow or Cardiac Stem Cells?

Oldroyd, Keith G, Berry, Colin, Bartunek, Jozef

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 239 KB

english, 2012

251

Evaluation of Residual Promoter Activity in γ-Retroviral Self-inactivating (SIN) Vectors

Xu, Wenqin, Russ, Jill L, Eiden, Maribeth V

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 322 KB

english, 2012

252

Correction of Murine Bernard–Soulier Syndrome by Lentivirus-mediated Gene Therapy

Kanaji, Sachiko, Kuether, Erin L, Fahs, Scot A, Schroeder, Jocelyn A, Ware, Jerry, Montgomery, Robert R, Shi, Qizhen

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 466 KB

english, 2012

253

Replication-Competent Retroviruses in Gene-Modified T Cells Used in Clinical Trials: Is It Time to Revise the Testing Requirements?

Bear, Adham S, Morgan, Richard A, Cornetta, Kenneth, June, Carl H, Binder-Scholl, Gwendolyn, Dudley, Mark E, Feldman, Steven A, Rosenberg, Steven A, Shurtleff, Sheila A, Rooney, Cliona M, Heslop, Hele

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 239 KB

english, 2012

254

SHAPE-directed Discovery of Potent shRNA Inhibitors of HIV-1

Low, Justin T, Knoepfel, Stefanie A, Watts, Joseph M, ter Brake, Olivier, Berkhout, Ben, Weeks, Kevin M

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 407 KB

english, 2012

255

Stanniocalcin-1 Rescued Photoreceptor Degeneration in Two Rat Models of Inherited Retinal Degeneration

Roddy, Gavin W, Rosa Jr, Robert H, Youn Oh, Joo, Ylostalo, Joni H, Bartosh, Thomas J, Choi, Hosoon, Lee, Ryang Hwa, Yasumura, Douglas, Ahern, Kelly, Nielsen, Gregory, Matthes, Michael T, LaVail, Matth

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 655 KB

english, 2012

256

Secondary Sphere Formation Enhances the Functionality of Cardiac Progenitor Cells

Cho, Hyun-Jai, Lee, Ho-Jae, Youn, Seock-Won, Koh, Seok-Jin, Won, Joo-Yun, Chung, Yeon-Ju, Cho, Hyun-Ju, Yoon, Chang-Hwan, Lee, Sae-Won, Lee, Eun Ju, Kwon, Yoo-Wook, Lee, Hae-Young, Lee, Sang Hun, Ho,

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 9.16 MB

english, 2012

257

Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene

van Til, Niek P, de Boer, Helen, Mashamba, Nomusa, Wabik, Agnieszka, Huston, Marshall, Visser, Trudi P, Fontana, Elena, Poliani, Pietro Luigi, Cassani, Barbara, Zhang, Fang, Thrasher, Adrian J, Villa,

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 3.31 MB

english, 2012

258

Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A

Bu, Jie, Ashe, Karen M, Bringas, John, Marshall, John, Dodge, James C, Cabrera-Salazar, Mario A, Forsayeth, John, Schuchman, Edward H, Bankiewicz, Krystof S, Cheng, Seng H, Shihabuddin, Lamya S., Pass

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 905 KB

english, 2012

259

Targeting TCTP as a New Therapeutic Strategy in Castration-resistant Prostate Cancer

Baylot, Virginie, Katsogiannou, Maria, Andrieu, Claudia, Taieb, David, Acunzo, Julie, Giusiano, Sophie, Fazli, Ladan, Gleave, Martin, Garrido, Carmen, Rocchi, Palma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.61 MB

english, 2012

260

rAAV-Mediated Tumorigenesis: Still Unresolved After an AAV Assault

Valdmanis, Paul N, Lisowski, Leszek, Kay, Mark A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 372 KB

english, 2012

261

Expression of Immunomodulatory Neutrophil-activating Protein of Helicobacter pylori Enhances the Antitumor Activity of Oncolytic Measles Virus

Iankov, Ianko D, Allen, Cory, Federspiel, Mark J, Myers, Rae M, Peng, Kah Whye, Ingle, James N, Russell, Stephen J, Galanis, Evanthia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 501 KB

english, 2012

262

Off-the-shelf Adenoviral-mediated Immunotherapy via Bicistronic Expression of Tumor Antigen and iMyD88/CD40 Adjuvant

Kemnade, Jan Ole, Seethammagari, Mamatha, Narayanan, Priya, Levitt, Jonathan M, McCormick, Alison A, Spencer, David M

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 469 KB

english, 2012

263

Sustaining Cardiac Claudin-5 Levels Prevents Functional Hallmarks of Cardiomyopathy in a Muscular Dystrophy Mouse Model

Delfín, Dawn A, Xu, Ying, Schill, Kevin E, Mays, Tessily A, Canan, Benjamin D, Zang, Kara E, Barnum, Jamie A, Janssen, Paul ML, Rafael-Fortney, Jill A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 477 KB

english, 2012

264

Advances in Foamy Virus Vector Technology and Disease Correction Could Speed the Path to Clinical Application

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 256 KB

english, 2012

265

Replication-competent Herpes Simplex Virus Retargeted to HER2 as Therapy for High-grade Glioma

Gambini, Eleonora, Reisoli, Elisa, Appolloni, Irene, Gatta, Valentina, Campadelli-Fiume, Gabriella, Menotti, Laura, Malatesta, Paolo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 5.29 MB

english, 2012

266

Corrigendum: Treatment of Brain Inflammatory Diseases by Delivering Exosome Encapsulated Anti-Inflammatory Drugs From the Nasal Region to the Brain

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 43 KB

english, 2012

267

A Designed Zinc-finger Transcriptional Repressor of Phospholamban Improves Function of the Failing Heart

Journal:

Molecular Therapy

Year:

2012

File:

PDF, 108 KB

2012

268

416. Co-Encapsulation of Islets with Mesenchymal Stem Cells Improves Islet Function

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

269

2. A Lentiviral Vector Pseudotyped with a Baboon Retrovirus Envelope Glycoprotein Outperforms VSV-G-LVs for Gene Transfer into Hematopoietic Stem Cells and Resting Lymphocytes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

270

3. Accessing the Unmappable Genome for Viral Integration Site Analysis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 445 KB

english, 2012

271

4. Histone Deacetylases Hinder Transgene Expression from Non-Integrating Lentiviral Vectors in Dividing and Non-Dividing Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 410 KB

english, 2012

272

5. In Vivo Mouse Models for Vector Genotoxicity Testing and Lentiviral Vector-Based Cancer Gene Discovery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 437 KB

english, 2012

273

6. In Vivo Gene Delivery to the Striatum Using a Novel Entry-Targeted Lentivirus: An Entry Restricted Lentivirus Vector for Neurodegenerative Diseases

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 194 KB

english, 2012

274

7. Comparison of Different Internal Promoter Performance in the CL20 SIN HIV Lentivector Used To Correct Human X-Linked Chronic Granulomatous Disease as Evaluated Ex Vivo and in NSG Mice Transplanted with Transduced Patient CD34+ Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

275

8. A Novel Lentiviral Pseudotype for Vascular Targeted Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

276

9. Identification of the Galactose Binding Domain of the AAV9 Capsid

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

277

10. A Comprehensive Analysis of Structural and Functional Significance of the 381 Amino Acids in the C-Terminal Half of AAV9 Capsid

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 210 KB

english, 2012

278

11. The 3D Structure of Adeno-Associated Virus Serotype 9 and Its Unique Properties

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

279

12. Microfluidic Live-Neuron Tracking of AAV9 Axonal Transport within the Endosomal System

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

280

14. Critical Amino Acid Redisues Contribute to Crossing Vascular Barrier

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

281

16. Role of AAVHSC15 Capsid Elements in In-Vivo Liver Transduction

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

282

17. Mass Spectrometry Identification of Proteins That Bind to Trafficking Plasmids during Transfection

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

283

18. Development of Vaxfectin®-Formulated HSV-2 Plasmid DNA Vaccines for Prophylactic and Therapeutic Applications

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 229 KB

english, 2012

284

19. In Vivo Structure-Activity Relationships for PEGylated Polyacridine Peptide Polyplexes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

285

20. Modified HK siRNA Polyplexes with Improved Pharmacokinetics Enhance Tumor Inhibition

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

286

22. Theoretical Model of Cationic Nanoparticles Dellivery and Margination under Convective Flow

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 291 KB

english, 2012

287

23. Facial Amphipathic Deoxycholic Acid-Modified Polyethyleneimine Used as SHP-1 siRNA Carrier for the Treatment of Ischemia-Reperfusion Cardiac Injury

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 310 KB

english, 2012

288

24. Pluronic Block Copolymer as a Potential Gene Delivery Candidate

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 187 KB

english, 2012

289

25. Dramatic Phenotypic Improvement in a Feline Model of Sandhoff Disease Treated by Adeno-Associated Virus Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 187 KB

english, 2012

290

26. Rescue of Lethal Phenotype in Aspartoacylase Knockout Mouse by Single Intravenous Delivery of Therapeutic Vector That Also Improves Neuropathology, Nephropathy, Motor Function and Visual Acuity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

291

27. More Pleiades MiniPromoters for the Brain and Eye

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

292

28. Systemic Delivery of rAAV9-hNAGLU in Adult Non-Human Primates: Efficient CNS and Somatic Transduction with No Detectable Toxicity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 215 KB

english, 2012

293

29. Noninvasive, Quantitative Imaging of Vector-Mediated D2R80A Reporter Gene Expression in the Living Animal Brain

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 188 KB

english, 2012

294

30. Presymptomatic Gene Therapy Cures Feline GM1 Gangliosidosis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 188 KB

english, 2012

295

31. Gene Therapy of Adult MLD Model Mice by Intrathecal Administration of Type 9 AAV Vector

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

296

32. Normalized Survival and Permanent Restoration of NAGLU Activity in the CNS, PNS and Somatic Tissues in MPS IIIB Mice after a Single Intravenous rAAV9-hNAGLU Gene Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

297

34. Demonstration of Systemic Exon 45-55 Multiple Skipping in Dystrophic mdx52 Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

298

35. RNAi Therapy for Dominant Limb Girdle Muscular Dystrophy Type 1A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

299

36. AAV-MTM1 Prolongs Survival and Rescues Severe Muscle Weakness in Mouse and Canine Models of X-Linked Myotubular Myopathy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 239 KB

english, 2012

300

37. Adjunctive β2-Agonist Therapy Enhances Biochemical Correction of Skeletal Muscle from an AAV Vector-Mediated Liver Depot in Advanced Pompe Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

301

38. Local Downregulation of Pre-B Cell Colony-Enhancing Factor (PBEF)/Visfatin Using an AAV5 Vector Encoding a Specific Small Hairpin RNA for PBEF Results in Amelioration of Arthritis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 345 KB

english, 2012

302

40. Long-Term Results of a Double-Blind, Placebo-Controlled, Multicenter Study Evaluating the Safety and Potential Activity of Escalating Doses of hMaxi-K Gene Transfer in Female Participants with Overactive Bladder and Detrusor Overactivity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 316 KB

english, 2012

303

41. microRNA 126 Is a New Regulator of Hematopoietic Stem Cells Homeostasis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 173 KB

english, 2012

304

42. Transfusion Independence and Rapid Engraftment of Nonhuman Primate Cord Blood Cells Expanded with the Combination of HOXB4 and Delta Ligand

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 173 KB

english, 2012

305

43. Xenotransplantation of iPSC Derived Neural Stem Cells from a Patient with Lysosomal Storage Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

306

44. Efficient Generation of Gene-Corrected, Vector- and Reprogramming Factor-Free iPS Cells from SCID-X1 Patients

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

307

45. Abrogation of EAE and Tolerance Induction of Naïve and Effector T Cells by Antigen Presenting Dendritic Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 209 KB

english, 2012

308

47. Robust Differentiation of Hematopoietic Progenitor Cells from Pigtail Macaque Induced Pluripotent Stem Cells toward Modeling Human Disease and Stem Cell Therapies In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

309

48. Derivation of “Semi-Universal Donor Stem Cells” That Express a Specific Class I Human Leukocyte Antigen (HLA) A0201

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

310

49. Transgene T Cell Response in a Phase 2 Clinical Trial of rAAV1.Alpha-1 Antitrypsin Gene Therapy Vector: CD8+ Mediated and HLA C*05 Restricted

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

311

50. Integration Site Analysis in a Clinical Trial of Lentiviral Vector-Based HSC Gene Therapy for MLD Shows High Levels of Polyclonal Hematopoietic Reconstitution and No Signs of Genotoxicity at One Year after Transplant

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 240 KB

english, 2012

312

51. Results of a Phase I Trial of SGT-53: A Systemically Administered, Tumor-Targeting Immunoliposome Nanocomplex Incorporating a Plasmid Encoding wtp53

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 222 KB

english, 2012

313

52. Increases in CD4 Counts and Effects on HIV in Aviremic HIV-Infected Subjects Infused with Zinc Finger Nuclease (ZFN) CCR5 Modified Autologous CD4 T-Cells (SB-728-T)

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 242 KB

english, 2012

314

53. Update on the U.S. Clinical Gene Therapy Trial for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID)

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

315

54. Administration of TGFβ-Resistant Tumor-Specific Cytotoxic T Lymphocytes (CTL) to Patients with EBV-Associated Hodgkin's Lymphoma (HL) and Non-Hodgkin Lymphoma (NHL)

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 247 KB

english, 2012

316

55. Long-Term Transgene Expression and Biological Effect Following IM Administration of AAV1-LPLS447X to LPL Deficient Patients

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 228 KB

english, 2012

317

56. ProSavin® a Gene Therapy Approach for Parkinson's Disease: Phase I Clinical Trial Update

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 228 KB

english, 2012

318

58. Designer Nuclease Platform Comparison: ZFNs Versus TALENs

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

319

60. Extrinsic Determinants of ZFN and TALEN-Mediated Gene Disruption

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

320

62. Evaluating the Safety of PiggyBac Mediated Gene Transfer in Human Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 271 KB

english, 2012

321

63. High-Level, Long-Term Gene Expression in Mice after Hydrodynamic Delivery of Sleeping Beauty Transposon/SB100x Transposase Combinations across a Surprisingly Broad Dose Range

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 255 KB

english, 2012

322

64. Genetic Correction of Dystrophin by Engineered Nucleases

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 278 KB

english, 2012

323

67. Targeted Integration in Human Epidermal Stem Cells by Zinc-Finger Nuclease-Mediated Homologous Recombination

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 175 KB

english, 2012

324

68. Immune Responses to Factor VIII Following Overexpression by Liver-Directed AAV Gene Therapy Do Not Correlate with Hepatic Cellular Stress Markers

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 175 KB

english, 2012

325

69. Results from the Long-Term Follow-Up of Severe Hemophilia B Subjects Previously Enrolled in a Clinical Study of AAV2-FIX Gene Transfer to the Liver

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 222 KB

english, 2012

326

70. Pancreatic Gene Delivery for Diabetes Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

327

71. Small Molecule Inhibitor of FAH Enzyme Allows for Selection of Genetically Resistant Donor Hepatocytes in Wildtype Settings In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

328

72. AAV2/2-CBA-REP1 Vector with WPRE Provides Functional Expression of the Transgene in Choroideremia Mouse Knock-Out and Patient Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 194 KB

english, 2012

329

73. Allele-Specific Silencing of Mutant Huntingtin in HD Neural Stem Cells and In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 194 KB

english, 2012

330

76. Identifying Motor Neuron Transduction Efficiencies That Are Efficacious in SMA Mice and Achievable by Intrathecal Delivery in a Large Animal Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

331

77. Anti-Cocaine Vaccine dAd5GNE Blocks Cocaine from Binding to the CNS Dopamine Transporter Critical to the Dopamine “Addiction” Pathway

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 187 KB

english, 2012

332

78. Overexpression of Non-Aggregatable Alpha-Synuclein Protects Against Human Wildtype Alpha-Synuclein Mediated Toxicity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 187 KB

english, 2012

333

79. Overexpression of the NDUFA6 Subunit of Complex I Ameliorates Neurodegeneration in Experimental Optic Neuritis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

334

80. Dose-Response Study of Continuous DOPA Delivery with AAV Gene Therapy Reveals Threshold and Kinetics for Restoration of Motor Function in a Rodent Model of Parkinson's Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

335

81. Targeting Castration Resistant Prostate Cancer (CRPC) with Autologous PSMA-Directed Chimeric Antigen Receptor T Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

336

82. Overcoming Tumor Immune Escape Using Bi-Specific CAR T Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

337

83. Early Acute Phase Response and Distribution of pbi-shRNA PDX-1 Lipoplex in Yucatan Swine

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

338

84. T-Cell Targeting an Ancient Retrovirus Expressed in Melanoma Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 356 KB

english, 2012

339

85. SNS01-T, an eIF5A-Based Gene Therapy Nanoparticle Designed for the Treatment of Multiple Myeloma, Has Anti-Tumoral Activity in Lymphoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 321 KB

english, 2012

340

86. A Highly Compact Epitope-Based Marker Suicide Gene for Safer and Easier Adoptive T-Cell Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 562 KB

english, 2012

341

87. Preclinical and Clinical Evaluation of Oncolytic Immunotherapy with Ad5/3-E2F1-Δ24-GMCSF (CGTG-602), a GM-CSF Producing Adenovirus Targeted to Tumors on Four Levels

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 401 KB

english, 2012

342

88. A Phase II Clinical Trial of Recombinant Human Endostatin Adenovirus (E10A) in Combination with Paclitaxel and Cisplatin in Patients with Head and Neck Carcinomas

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 428 KB

english, 2012

343

89. Image-Guided, Lobe-Specific Hydrodynamic Gene Delivery to Baboon Liver

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

344

90. Long-Term Elimination of Human Hepatitis C Virus in Human Hepatocyte Chimeric Mice after Single Administration of Plasmid DNA Expressing Human Interferon-γ

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

345

91. Enhancing Gene Transfection In Vivo Using Therapeutic Ultrasound and New Microbubbles

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 239 KB

english, 2012

346

92. Gene Electrotransfer to a Beating Heart in a Porcine Ischemia Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 176 KB

english, 2012

347

93. Molecular Cardiac Surgery Mediated ssAAV1-SERCA2a Gene Therapy Restores Global and Regional Contractility in Ovine Ischemic Cardiomyopathy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 316 KB

english, 2012

348

94. Identification of Novel Cardioprotective Factors by In Vivo Functional Gene Selection Using AAV Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 299 KB

english, 2012

349

95. Anti-Hypertrophic Actions of Cardiac Expression of a GC-A Agonist and a Novel Dual GC-A and GC-B Agonist in a Rat Model of Hypertensive Heart Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 299 KB

english, 2012

350

96. Combined Gene- and Drug-Based Intervention Is Protective Following Cerebral Ischaemia/Reperfusion

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 392 KB

english, 2012

351

97. An Optimized Two-Finger Archive for Efficient ZFN-Mediated Gene Targeting

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 425 KB

english, 2012

352

98. Genome Wide Characterization of Aberrant Splicing Events Induced by Lentiviral Vector Integrations

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

353

99. Development of a High-Through Approach for Quantitating Cellular microRNA Activity and Identifying Optimal Target Sites for microRNA-Based Vector Targeting

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

354

100. The Long Noncoding RNA, Fas-Antisense or Saf, Is Involved in Human Erythropoiesis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 251 KB

english, 2012

355

102. Chemically Modified mRNA Encoding mFoxp3 Protects Against Airway Inflammation in a Murine Model of Allergic Asthma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 243 KB

english, 2012

356

103. Nectin-4 Is the Measles Virus Host Exit Receptor: Implications for Other Respiratory Infections and for Oncolysis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

357

104. Adeno-Associated Virus-Directed Persistent Expression of an Anti-Nicotine Antibody Gene for Smoking Cessation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

358

105. Identification of Novel Genes Regulating AAV Transduction by High-Throughput, Genome-Wide siRNA Screening

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

359

106. microRNA Regulated Cardiotropic AAV Vectors with Skeletal Muscle and Liver Specific Suppression of Transgene Expression

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

360

107. A Potential Functional Domain Rich in Basic Amino Acids within the AAV2 Assembly-Activating Protein (AAP) Constrains the Structural Diversity of the AAV2 Capsid

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 208 KB

english, 2012

361

109. High-Throughput Small Molecule Screen Yields Novel Classes of Compounds That Modulate Adeno-Associated Virus Transduction

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

362

110. Persistent Transgene Expression from AAVHSC15 in Mice Pre-Treated with High Dose Human IVIG

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 176 KB

english, 2012

363

112. Design and Characterization of New Generation Synthetic AAV Capsid Libraries

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 176 KB

english, 2012

364

113. Characterization of Porcine Adeno-Associated Viruses Belonging to Unique Clades

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 230 KB

english, 2012

365

114. Limitations of Encapsidation of Recombinant scAAV2 Genomes in Different Serotype Capsids and Their Quantitation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 213 KB

english, 2012

366

115. Bio-Engineering of Adeno-Associated Virus Serotype (AAV)-2 Capsid at Serine/Threonine Residues Improves Its Transduction Efficiency Both In Vitro and In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 357 KB

english, 2012

367

116. Site-Directed Mutagenesis of Surface-Exposed Tyrosine, Serine, and Threonine Residues Leads to High-Efficiency Transduction by Recombinant Adeno-Associated Virus 2 Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 312 KB

english, 2012

368

118. High-Efficiency Transduction of Primary Human Hematopoietic Stem Cells by Optimized AAV6 Serotype Vectors and Erythroid Lineage-Restricted Transcriptional Targeting Using the Human Parvovirus B19 Promoter

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 357 KB

english, 2012

369

119. In Vivo Gene Delivery Using Microvesicle-Associated AAV

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

370

120. Characterization of the Role of Vaccinia Virus in the Activation of Adeno-Associated Virus Promoters

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

371

121. Sleeping Beauty Transposon-Based Integration System Analysis in Human Epithelial Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 232 KB

english, 2012

372

122. In Vivo Selection of Hepatocytes Engineered To Express CrmA Using the Sleeping Beauty Transposon System

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

373

123. Enhancement of Transfection of Linear Expression Cassettes after Ligation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

374

124. Human Artificial Chromosome (HAC) Vector with a Conditional Centromere for Correction of Genetic Deficiencies in Human Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

375

125. Novel Antitumor Strategy by Transfection of Plasmids Encoding Pathogenic Antigen- and Cytokine-Genes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

376

126. Zinc Finger Nuclease-Mediated Transgenesis in Human Cord-Derived Cells: En Route to Cell Therapy for Hemophilia A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

377

127. PROGNOSE: An Online Tool for Predicting and Analyzing the Off-Target Site Effects of Designer Nucleases

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

378

128. Implementation of a Nuclear Factor Kappa B DNA Nuclear Targeting Sequence Improves In Vitro Suicide Gene Therapy Efficacy in Both Small- and Non-Small Cell Lung Cancer Cell Lines

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

379

129. Stimulation of Homologous Recombination-Mediated Genome Editing through Donor DNA Processing

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

380

130. Safe Harbor Locus Preservation with Zinc-Finger Nuclease Mediated Gene Addition in Mouse Primary Fibroblasts

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 232 KB

english, 2012

381

131. Systematic Identification of Bi-Functional Short-Hairpin RNA (shRNA) Specific for Single-Nucleotide KRAS Mutations

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

382

132. Prevention of Hepatocellular Adenoma and Correction of Metabolic Abnormalities in Murine Glycogen Storage Disease Type Ia by Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 376 KB

english, 2012

383

133. Survival with Normal Neurological Development of the Juvenile Lethal Urea Cycle Defect Arginase Deficient Mouse with AAV Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 337 KB

english, 2012

384

134. Gene Therapy Versus Enzyme Replacement Therapy in a Murine Model of Mucopolysaccharidosis VI

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 354 KB

english, 2012

385

135. Safety of Liver-Directed AAV2/8-Mediated Gene Transfer in a Large Animal Model of Lysosomal Storage Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

386

136. Increased Frequency of Permanent Integration Following Liver-Directed Gene Therapy with rDNA-AAV2/8 In Murine Phenylketonuria

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

387

137. Neonatal Gene Therapy for OTC Deficiency

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

388

138. Liver Gene Therapy for PKU Using Naked DNA/Minicircle Vectors Expressing Phenylalanine Hydroxylase from a Synthetic Minigene

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

389

139. In Vivo Gene Transfer of Different Isoforms of the MeCP2 (Rett Syndrome) Gene Is Associated with Very Different Toxicity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 226 KB

english, 2012

390

140. Sex-Dependent Expression of Very Long Chain acyl-CoA Dehydrogenase (VLCAD) Transgene and the Role of the Liver in Biochemical and Phenotypic Correction of VLCAD Deficiency

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

391

141. Human Versus Murine Phosphoglycerate Kinase (PGK) Promoter/Enhancer in Canine Leukocyte Adhesion Deficiency

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

392

142. Direct Hepatic Injections of HDAd Vector Are More Efficient and Safer Than Systemic Intravenous Injections for Liver-Directed Gene Therapy of Crigler-Najjar Syndrome Type I

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

393

143. Pharmacological Read-Through of Nonsense ARSB Mutations as a Potential Therapeutic Approach for Mucopolysaccharidosis VI

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

394

144. A Murine Model of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

395

145. Demonstration of Pre-Clinical Efficacy of rAAV8 Gene Transfer as a Treatment for Methylmalonic Acidemia Using a Vector Suitable for Human Clinical Trials

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

396

146. Hepatocyte Therapy for MUT Methylmalonic Acidemia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

397

147. In Vitro and In Vivo Delivery of Surface Immobilized AAV-2/9 to Arterial Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

398

148. Hydrodynamic Gene Transfer of a Cox-1/ Prostacyclin Synthase Fusion Protein Results in Endogenous Production of Supra-Therapeutic Levels of Prostacyclin in Rats

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 233 KB

english, 2012

399

149. Process Development for Manufacture of hES Cell-Derived Cardiomyocytes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

400

150. Osteopontin Treated Circulating Angiogenic Cells Enhance Functional Recovery in a SCID Model of Hindlimb Ischemia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

401

151. Liposomal Surface Coatings of Metal Coronary Artery Stents for Efficient Non-Viral Gene Delivery to the Injured Vasculature

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 425 KB

english, 2012

402

152. The Effects of AdVEGF-B and AdVEGF-D on Angiogenesis and Arteriogenesis in a Novel Porcine Model of Percutaneous Bottleneck Stent Induced Chronic Myocardial Ischemia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 398 KB

english, 2012

403

153. GM-CSF Gene Therapy for Rabbit Acute Hind Limb Ischemia by Electroporation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 422 KB

english, 2012

404

154. Therapeutic Angiogenesis for Limb Ischemia: Combined Approach Using Novel Plasmid Vectors and Gene-Modified Adipose Derived Stromal Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

405

155. Neutralizing Antibodies Against AAV Serotypes 1, 2, 6 and 9 in Sera of Commonly Used Animal Models and Their Significance for In Vivo Experiments

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

406

156. Interleukin-35 Gene Therapy Alleviated Cardiac Allograft Rejection in Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 306 KB

english, 2012

407

157. Progeny of Pronuclear Injections of Mutant Human Mitochondrial Genes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 282 KB

english, 2012

408

158. Evaluating Huntington's Disease Therapeutics In Vivo Using a Sensitive Measure of Transcriptional Dysregulation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 288 KB

english, 2012

409

159. Long-Term Rescue by AAV-Delivered Therapy in Autosomal Dominant Retinitis Pigmentosa (ADRP) Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

410

161. Rapid Transgene Expression in Multiple Precursors Cell Types of Adult Rat Subventricular Zone Mediated by Adeno-Associated Type 1 Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 653 KB

english, 2012

411

162. AAV Mediated Gene Delivery Suppresses Inflammatory Response Associated with GM2 Gangliosidosis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 638 KB

english, 2012

412

163. Delivery of a Therapeutic Molecule to the Brain for the Treatment of Neurodegenerative Diseases: Production of AAV-5-GDNF by a GMP-Compliant and Scalable Production Platform Results in a Highly Efficient Gene Therapy Vector

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 251 KB

english, 2012

413

165. Natural Killer Cells for Therapy of Neural Tumors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 329 KB

english, 2012

414

166. Reversal of Pathology in CHMP2B-Mediated Frontotemporal Dementia Patient Cells Using Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 264 KB

english, 2012

415

167. Alpha-Synuclein Overexpression in the Aged Rat Brain: Is the Aging Nigrostriatal System More Suseptible to α-Synuclein Mediated Neurodegeneration?

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 264 KB

english, 2012

416

168. Increased Longevity of Rescue of Light-Induced Retinal Damage in an Adult Mouse Using Peptide for Ocular Delivery (POD) as a Gene Transfer Vector

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 281 KB

english, 2012

417

169. Developing Engineered Zinc Finger Repressors of Huntingtin as a Potential Therapy for Huntington's Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 187 KB

english, 2012

418

170. Improved Neuroplasticity and Decreased Apoptosis by Intralesional Transplantation of Mesenchymal Stem Cells after Severe Spinal Cord Injury in Rats

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 187 KB

english, 2012

419

171. Repair of Spinal Cord Injury (SCI) Using Monocytes Transfected with Adenoviral Vector Expressing Decorin in a Rat SCI Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 206 KB

english, 2012

420

172. Muscle Force Improvement by Long-Term Systemic Expression of an AAV9 Minidystrophin after Delivery in Young Adult GRMD Dogs without Immune Suppression

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 181 KB

english, 2012

421

173. RNA Interference Inhibits DUX4-Induced Muscle Toxicity In Vivo: Implications for a Targeted FSHD Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 181 KB

english, 2012

422

174. A Novel Rationally Designed AAV Micro-Utrophin Vector Recruits nNOS to the Sarcolemma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 306 KB

english, 2012

423

175. Determining the Therapeutic Window: Central Nervous System Administration for Vector Mediated Gene Replacement in a Severe Model of Spinal Muscular Atrophy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 306 KB

english, 2012

424

178. Increased Overexpression of Human Alpha7 Integrin as a Potential Therapy for Duchenne Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

425

179. Systemic Delivery of nNOS-Recruiting Mini-Dystrophin AAV Vectors Ameliorated Muscular Dystrophy in a Mouse DMD Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 199 KB

english, 2012

426

180. Adeno Associated Virus (AAV) Mediated Follistatin Gene Transfer Toxicology Studies in Preparation of Phase I/II Clinical Trial

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 199 KB

english, 2012

427

181. Abbreviated Dystrophins Restore the Passive Properties of the Extensor Digitorum Longus Muscle in Dystrophin-Null Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 199 KB

english, 2012

428

182. A Novel Cell Therapy for Muscular Dystrophy by Bone Marrow Stromal Cell: Mesenchymal Stem Cell Derived from Bone Marrow Can Affect Skeletal Muscle Regeneration

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 409 KB

english, 2012

429

183. Identification of Decorin Derived Peptides with a Zn2+ Dependent Anti-Myostatin Activity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 378 KB

english, 2012

430

185. AAV Micro-Dystrophin Therapy Alleviates Stress-Induced Cardiac Death but Does Not Reduce Myocardial Fibrosis in >21-m-Old mdx Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 422 KB

english, 2012

431

186. Myogenic Transduction and Cell Surface Marker Selection of DMD Fibroblasts Enable Stable Dystrophin mRNA Expression for Exon Skipping Assay

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

432

187. Randomized Phase II Trial of Post-Operative Adjuvant Chemotherapy ±FANG™ Autologous Tumor Cell Vaccine in Colorectal Carcinoma with Liver Metastases

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

433

188. Hematopoietic Stem Cell Immunotherapy for Melanoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 228 KB

english, 2012

434

189. CD40-Ligand Armed Vaccinia Virus for Induction of Anti-Tumor Immune Response

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

435

191. Amelioration of Colitis and Its Associated Colorectal Tumors by CEA — Redirected Tregs

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 248 KB

english, 2012

436

192. Naive-Derived Memory Stem T Cells: A Novel Promising Platform for Cancer Immune-Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 232 KB

english, 2012

437

193. Engineering CD20-Specific Chimeric Receptor-Redirected T Cells with Inducible Co-Expression of a Caspase-9 Based Suicide Switch for Adoptive Immunotherapy of CD20 Positive Lymphoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 232 KB

english, 2012

438

194. Combining the Dual Tyrosine Kinase Inhibitor, Lapatinib, with HER2 Specific Cytotoxic T-Lymphocytes in the Treatment of HER2 Overexpressing Breast Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

439

195. Development of a CAR T Cell Platform with Dissociated Signalling Domains for Selective Reactivity Against Ovarian Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

440

196. Upregulation of NKG2D Ligands by Sodium Valproate (VPA) Increased the Susceptibility of Cancer Cells That Are Recognized by Chimeric NKG2D-4-1BB-CD3z T Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

441

197. AdCD40L Reduces the Presence of Myeloid-Derived Suppressor Cells in the Tumor Microenvironment

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

442

200. Effect of γ34.5 Deletions on Oncolytic Herpes Simplex Virus Activity in Glioblastoma Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

443

201. Systemic Cancer Virotherapy with MDVV, a Combined miRNA-Regulated and Thymidine Kinase-Deleted Oncolytic Vaccinia Virus

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

444

203. A New Class of Glioblastoma Restricted oHSV Replication Based on microRNA Control of Essential Virus Gene Expression

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

445

205. Combined Therapies with Oncolytic Adenovirus Suppress Tumor Growth in Hamsters

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 236 KB

english, 2012

446

206. microRNA-Sensitive and Armed Oncolytic Measles Virus for Therapy of Pancreatic Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

447

207. Tat-PTD-Modified Oncolytic Adenovirus Driven by the SCG3 Promoter and ASH1 Enhancer for Neuroblastoma Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

448

208. Modulation of Complement System for Enhancing Oncolytic Vaccinia Virus-Mediated Cancer Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 389 KB

english, 2012

449

210. Midkine Promoter-Driven Oncolytic Adenovirus with Ad35 Fiber Modification Achieves Enhanced Transduction of Human Malignant Mesothelioma Cells In Vitro and In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 373 KB

english, 2012

450

211. Efficacy and Safety of Gene-Chemo Therapy Using Oncolytic Adenovirus Expressing Uracil Phosphoribosyl Transferase (UPRT) Followed by 5-Fluorouracil Administration in Syngenic Pancreatic and Biliary Cancers in Immunocompetent Adenovirus-Permissive Syrian Hamsters

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

451

212. Phase I Trial of Intratumoral (IT) Administration of HF10, a Replication-Competent Herpes Simplex Virus Type 1, in Patients with Refractory Superficial Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

452

213. [F18]-FDG-PET Versus CT for Evaluation of Oncolytic Virus Treatment in Advanced Cancer Patients

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

453

214. Prolonged Survival of Mice in an Orthotopic Glioblastoma Stem Cell Mouse Model after Treatment with an Y-Box Binding Protein 1 (YB-1) Dependent Oncolytic Adenovirus

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

454

215. Preclinical Development of ORCA-010, a Novel Potency-Enhanced Oncolytic Adenovirus

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

455

216. PEGylation of Vesicular Stomititis Virus (VSV) Extends Virus Persistence in Blood in the Presence of Neutralizing Antibodies

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

456

217. Development of Efficient Oncolytic Adenoviruses Against Melanoma by Combining Rational Engineering and Directed Evolution

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

457

218. Inducible Caspase 9 as Safety Switch in Gene Therapies Targeting Hematopoietic Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 219 KB

english, 2012

458

219. Tracking the Fate and Activity of Individual HSC and Memory Stem T Cell Clones in GT Patients through Insertional Tagging

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

459

220. Integration Site Analysis of Wiskott-Aldrich Syndrome Patients Treated with Lentiviral Vector Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

460

221. Efficacy and Safety of the Foamy Viral Vector ΔΦMscvCD18 in Long-Term Follow-Up (4-6 years) of Four Dogs with Canine Leukocyte Adhesion Deficiency

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

461

223. Lentiviral Vector Mediated Gene Therapy for X-Linked Lymphoproliferative Disease Restores Humoral and Cellular Functions

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

462

224. Transduction of Human Bone Marrow CD34+ Cells by a Lentiviral Vector Carrying a Modified Human Beta-Globin Gene for Sickle Cell Disease Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

463

225. Development of a Clinically-Applicable Lentiviral Vector for RS-SCID Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 227 KB

english, 2012

464

226. Complete Long-Term Correction of Hemophilic B Mice Using Integration-Defective Lentiviral Vectors Expressing Codon-Optimized R338L Human Factor IX

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

465

227. Engineered Nuclease Mediated Gene Targeting of the Human IL2Rγ Gene

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

466

228. AAV Vector and Transgene Modifications for Improved Efficacy in Hemophilia Gene Therapy with Continuous Expression of FVIIa

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

467

229. Vectofusin-1 Augments Lentiviral Transduction of Human Hematopoietic Cells by Enhancing Adhesion and Fusion of Particles to CD34+ Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

468

230. Correction of Immunity in Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-Optimized RAG2 Therapeutic Transgene

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 342 KB

english, 2012

469

232. Alpha-1 Antitrypsin (AAT) Expression 1 Year after IM Administration of rAAV1-CB-hAAT in a Phase 2 Clinical Trial

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 334 KB

english, 2012

470

233. rAAV9 Airway Delivery Results in Effective Knockdown of Mutant alpha 1-Antitrypsin in the Liver While Upregulating Wildtype alpha 1-Antitrypsin in the Lung

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 208 KB

english, 2012

471

234. shRNA for Thymic Stromal Lymphopoietin as an Novel Antagonist for Pulmonary Fibrosis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 208 KB

english, 2012

472

235. Manipulation of Airway Epithelia To Improve siRNA Delivery and RNAi Efficacy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

473

236. Gene Therapy of Cystic Fibrosis: Impact of Plasmid DNA on In Vivo Lipid-Based Transfection Efficacy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

474

237. Lentiviral Vector Gene Transfer to Porcine Sinus and Pulmonary Airways

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

475

238. Enhancing Immunotherapy with a Replicating Vaccinia Virus Expressing CXCL11 to Chemoattract T-Cells into Tumors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 600 KB

english, 2012

476

239. High Speed X-Ray Imaging Reveals Dosed Fluid Dynamics and Fate in Mouse Nasal and Lung Airways

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 565 KB

english, 2012

477

240. Genetically Engineered Measles Virus Edmonston Strain Expressing the Wild-Type N, P, L Genes (MV-NPL) Is a Promising Oncolytic Virotherapy Agent Against Lung Cancer Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

478

241. A Preclinical Model To Study Tumor-Infiltrating Lymphocyte (TIL) Anergy/Exhaustion in Adoptive T Cell Therapy (ACT) of Thoracic Malignancies

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

479

242. A Truncation Mutant of CFTR, Δ27-264 CFTR, Corrects Processing Mutants of CFTR

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 251 KB

english, 2012

480

243. Efficient Apical Transduction of Polarized Human Airway Epithelium by Adenovirus Serotype 3

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 231 KB

english, 2012

481

244. Synthetic DNA Vaccine Against Avian Influenza A Virus

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 231 KB

english, 2012

482

247. Comparative Analysis of the Transduction Efficiency of Five AAV Serotypes and Lentiviral Vectors in Lung Cancer Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

483

248. Tolerance Induction and Long-Term Correction in a Mouse Model of Hemophilia A with an AAV8 Serotype Carrying Codon-Optimized Human FVIII

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

484

251. AAV Capsid Antigen Cross-Presentation Is Dependent on Proteasomal Degradation and Nuclear Virion Uncoating

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

485

252. AAV Vector-Mediated Systemic Delivery of Interleukin-10 Attenuates the Progression of Peritoneal Fibrosis in a Rat Model of Peritoneal Dialysis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 250 KB

english, 2012

486

253. Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to Adeno-Associated Virus Vector Containing a CMV Enhancer/Chicken β-Actin Cassette in Murine Pompe Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 225 KB

english, 2012

487

254. Quantitative Imaging Comparison of Intracellular Trafficking of AAV Capsid Variants

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

488

255. Impact of Pre-Existing Neutralizing Antibodies (NAbs) on Expression of a SIV Antibody Following Intramuscular (IM) Administration of AAV2/8

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 251 KB

english, 2012

489

257. Characterization of Naturally-Occurring Humoral Immunity to AAV in Sheep

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 222 KB

english, 2012

490

258. Innate Immune Response Affects AAV-Mediated Hepatic Trangene Expression

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 248 KB

english, 2012

491

259. Prevalence and Correlation of Neutralizing Antibodies Against AAV2, 7, 8, 9 and RH32.33 in the Human Sera from Southern China

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

492

260. Epirubicin Potentiates rAAV2/5-Mediated TRAIL Expression in Fibroblast-Like Synoviocytes and Augments the Anti-Arthritis Effects of rAAV2/5-TRAIL

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

493

261. Nanoparticles as Delivery Systems for Antisense Oligoribonucleotides Aimed at Exon Skipping: Results in the Dystrophinopathic mdx Animal Model by Intraperitoneal Ad Oral Administration Routes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 434 KB

english, 2012

494

262. Preclinical Biodistribution, Toxicology and Survival Assessments for the Administration of pbi-shRNA™ STMN1 Lipoplex

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 398 KB

english, 2012

495

263. Aptamer-siRNA Chimera Inhibitors of Intimal Hyperplasia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 576 KB

english, 2012

496

264. Phase I Study of pbi-shRNA™ STMN1 Bilamellar Invaginated Vesicle (BIV) Lipoplex (LP) Via Intratumoral Injection in Advanced Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 337 KB

english, 2012

497

265. Nanoparticles with Triplex-Forming Oligonucleotides for Site-Specific Editing of the Human Cystic Fibrosis Transmembrane Receptor Gene

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 337 KB

english, 2012

498

266. Effect of Continuous Exposure to Antisense BNA Slowly Released in Intraperitoneal or Subcutaneous Tissue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 185 KB

english, 2012

499

267. Antiviral Potency of Cleaved and Uncleaved Anti-HIV RT Aptamers Expressed Intracellularly Via a Hammerhead Ribozyme Cassette

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 185 KB

english, 2012

500

268. Adenovirus Expressing Artificial microRNAs Inhibit the Replication of H1N1 Influenza A Virus in Infected Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 209 KB

english, 2012

501

269. Allele Specific Targeting of LRRK2 G2019S with shRNA-Induced RNA Interference as a Potential Gene Therapy for Parkinson's Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

502

271. Analysis of Growth and Invasion of Human Glioma Cell Lines after Gene Silencing of IL-13Ra2 In Vitro

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 222 KB

english, 2012

503

272. Synthetic Double-Stranded siRNA-Based Therapeutics Targeting nNOS for Prevention of 6-OHDA Neurotoxicity in Neuroblastoma Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

504

273. Synthetic Double-Stranded siRNAs Can Silence the Expression of Neuronal Nitric Oxide Synthase Enzyme (nNOS) from Neuroblastoma Cells in a Temporal and Dose Dependent Manner

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 240 KB

english, 2012

505

274. Eag1 Gene Silencing by Non-Viral Expression Vector Reduces the Viability of Neuroblastoma Cells in Culture

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

506

275. Time-Dependent siRNA Effects on nNOS Message in Glioma Cells in Culture

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

507

276. RNAi-Based Therapeutics Targeting nNOS for Prevention of Neuroblastoma Cell Apoptosis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 368 KB

english, 2012

508

278. Gene Therapy of Erythropoietic Porphyria Mice with Induced Pluripotent Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 414 KB

english, 2012

509

279. Derivation and Functional Analysis of Patient Specific Induced Pluripotent Stem Cells as an In Vitro Model of Chronic Granulomatous Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

510

280. Generation of Integration-Free iPSCs from an X-CGD Patient's Blood Cells as Clinically Relevant Target for Gene-Repair Using Designer ZFN or TALEN

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 556 KB

english, 2012

511

282. A Ubiquitous Chromatin Opening Element (UCOE) Prevents Transgene Silencing in Murine Pluripotent Cells and Their Differentiated Hematopoietic Progeny

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

512

283. Therapeutic Potential of Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitor Cells for Canavan Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

513

284. The Application of A9 Dopaminergic Neurons Derived from Human Parthenogenetic Stem Cells for the Treatment of Parkinson's Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

514

285. SOX2 Is a Possible Target for the Treatment of Tumors Emerged in the Process of Reprogramming for iPSC Generation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

515

286. In Vivo and In Vitro Characterization of Early Hepatocytes Derived from Human Parthenogenetic Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

516

287. Differentiation of Human Pluripotent Parthenogenetic and Embryonic Stem Cells into Cornea-Like Constructs for Transplantation and for Studying Ocular Drug Absorption In Vitro

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

517

288. Systematic Development and Qualification of Assays for the Characterization of Human Embryonic Stem Cells and Their Differentiated Progeny

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

518

291. Human Visual Cortex Response to Retinal Gene Therapy Re-Administration

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 606 KB

english, 2012

519

292. In Vivo Nanoparticle Delivery for Site-Specific Genome Editing of Hematopoietic Cells in a Humanized Mouse

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 591 KB

english, 2012

520

293. Delivery of Novel microRNAs Improves Cardiac Myocyte Proliferation In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 609 KB

english, 2012

521

294. De Novo Design of Tissue-Specific Regulatory Elements Results in Robust Transduction in Heart and Liver: Implications for Cardiovascular Disease and Hemophilia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

522

295. Type I IFN Signaling Induces Epigenetic Modification of Helper-Dependent Adenoviral Vector DNA and Silences Transgene Expression at Transcriptional Level in Liver after Systemic Administration

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 230 KB

english, 2012

523

297. Identification of a Novel Small Molecule Inhibitor of FX-Mediated Ad5 Gene Transfer for Gene Therapy Applications

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

524

298. Novel CpG-Rich Oncolytic Adenovirus Backbone To Stimulate Tumor-Specific Immune Response and Block the Suppression of Myeloid-Derived Suppressive Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

525

299. Strengthening of Antitumor Immune Memory and Prevention of Thymic Atrophy Mediated by Adenovirus Expressing IL-12 and GM-CSF

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

526

300. Systemic Delivery of Oncolytic Adenoviruses Targeting Transforming Growth Factor Beta Inhibits Established Bone Metastasis in a Prostate Cancer Mouse Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 540 KB

english, 2012

527

301. Viral Genome DNA/Lipoplexes Elicit In Situ Oncolytic Viral Replication and Potent Antitumor Efficacy Via Systemic Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 516 KB

english, 2012

528

302. Transposition from an Adenoviral Vector Shows a Strong Bias towards Somatic Integration into the X Chromosome

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 540 KB

english, 2012

529

303. Efficient Site-Specific Integration in Human Hematopoietic Stem and Progenitor Cells by Zinc Finger Nucleases and Integrase-Defective Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

530

304. Engineered Zinc Finger Nickases for Stimulating Homology-Directed Repair with Decreased Mutagenic Effects

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

531

306. Homologous-Recombination Mediated Genome Editing at the Adesonine Deaminase Locus in Patient-Derived Fibroblasts Using TAL Effector Nucleases

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

532

307. Gene Targeting of the Human Globin Loci Using Engineered Nucleases

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

533

308. Zinc-Finger Nuclease-Mediated Correction of RS-SCID

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

534

309. Site-Specific Gene Correction of β-Globin Using Zinc Finger Nucleases

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

535

310. High-Throughput Gene Editing in Human Cells Enabled by Fast Ligation-Based Automatable Solid-Phase High-Throughput (FLASH) Assembly of TALENs

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

536

311. Generation and Correction of a Novel Mouse Model of Acid Ceramidase Deficiency

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

537

312. Rapid CNS Correction in MPS IIIA Mice Using Systemic Delivery of Self-Complementary AAV9 and AAVrh74 SGSH Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 194 KB

english, 2012

538

313. Liver-Directed Gene Therapy for Mucopolysaccharidosis Type I (MPS I)

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 209 KB

english, 2012

539

314. Hematopoietic Stem Cell Gene Therapy with Lentiviral Vector in 4 Patients with Cerebral X-Linked Adrenoleukodystrophy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 175 KB

english, 2012

540

316. Integration Site Distribution of Lentiviral Vector in Gene Therapy for X-Adrenoleukodystrophy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

541

317. AAV Based Gene Therapy Rescues the Murine Cardiac Phenotype Associated with Friedreich's Ataxia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

542

318. Development of Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH) Due to Perforin Deficiency

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 245 KB

english, 2012

543

319. Phase I Study To Improve Virus-Specific Immune Reconstitution after Cord Blood Transplantation Using Cord Blood-Derived Virus-Specific T Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

544

320. HPV Specific CTLs Induced in CIN2/3 Patients after DNA Immunization Using Electroporation in a Phase I Trial

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

545

321. Genetic Modification of Stem Cells Leads to an Enhanced Immune Response and Reduced Viral Pathogenesis upon SHIV-Challenge in the Macaque Model of AIDS

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 243 KB

english, 2012

546

322. Development of a Highly Efficient Second Generation Nicotine-Conjugate Vaccine To Treat Nicotine Addiction

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 243 KB

english, 2012

547

323. Expression of Broadly Neutralizing Antibodies (NAbs) Against HIV in RAG KO Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 268 KB

english, 2012

548

324. DNA Vaccination To Generate Chikungunya Virus-Specific Immunity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 203 KB

english, 2012

549

325. Lentiviral Gene Therapy Against HIV-1 Using a Novel TRIM21-Cyclophilin A Fusion Restriction Factor

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 203 KB

english, 2012

550

327. New Safety and Efficacy Data of a Phase 1-2 Study in Patients with Advanced Renal Cell Carcinoma, Following Vaccination with Fourfold Gene-Modified, Allogeneic Tumor Cells Combined with a DNA-Based Immunomodulator

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

551

328. The Broad Antigenic Repertoire Provided by a VSV-cDNA Vaccine Forces Tumor Escape Variants into a Reproducible, and Predictable, Phenotype Which Is Highly Sensitive to Second Line Chemotherapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

552

329. Redirection of TH17 Cells with a Car Containing the ICOS Costimulatory Domain Enhances Function, Antitumor Activity and Persistence of TH17 Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

553

330. Tumor Pericytes: A Novel Target for Immunotherapy in Renal Cell Carcinoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

554

332. Sipuleucel-T in Metastatic Castrate-Resistant Prostate Cancer (mCRPC): Clinical Evidence of the Immunologic Mechanism of Action (MOA)

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

555

333. A Rationally Designed Oncolytic Adenovirus, Ad-TD-IL-12, Cures Pancreatic Cancer in the Immunocompetent Syrian Hamster

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

556

335. Safe and Durable Hepatic Expression of Anti-HCV shRNA in a Non-Human Primate Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 206 KB

english, 2012

557

336. Phase II Study of FANG™ (Autologous Tumor Cell — GMCSF/pbi-shRNA™ Furin DNA Plasmid) Vaccine in Advanced Melanoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 206 KB

english, 2012

558

337. Effect of Argonaute-2 in Mediating Non-Coding RNA Expression

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

559

338. Effective Regression of Melanoma Cancer by Delivery of Combinatorial siRNA Using an Arginine-Engrafted Biodegradable Polymer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

560

339. Detection Methods for In Vitro and In Vivo Characterization of Cell-Specific, Cell-Internalizing RNA Aptamers for siRNA Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

561

340. The Anti-Gene Oligonucleotide “Zorro-LNA”, Delivered by a Cationic Lipid, DownRegulates Huntingtin Gene Expression in Mammalian Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 290 KB

english, 2012

562

341. Activation of Latent HIV-1 by Targeted Suppression of an Endogenous HIV-1 Expressed Antisense Non-Coding RNA

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 263 KB

english, 2012

563

342. New Approaches for Targeted Delivery of Dicer-Substrate siRNA

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 263 KB

english, 2012

564

344. Optimization of the MCM7 miRNA Cluster as a Novel Platform to Multiplex Small Interfering and Nucleolar RNAs for Combinational HIV-1 Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

565

345. Lentiviral Vectors Targeting Human T Cells for RNAi-Mediated Gene Therapy of HIV Infection

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

566

346. Identification of a Domain Responsible for the Dose-Dependent Antiproliferative and Apoptotic Effects of Sleeping Beauty Transposase

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

567

347. Addition of a Modified polyA Signal and Intron into a Lentiviral Platform for RNAi and Protein Over-Expression Provides Potent Efficacy While Maintaining High Vector Titers

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

568

349. A Cell-Based Assay To Detect Rare Psi-Gag Recombinants in Lentiviral Vector Preparations

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 343 KB

english, 2012

569

350. Vector Design-Dependent Modulation of Viral-Cellular Chimeric Fusion Transcripts Derived from Self-Inactivating (SIN) Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 343 KB

english, 2012

570

351. Directed Integration of Insulated Lentiviral Vectors to the Heterochromatin towards Safer Gene Transfer to Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 368 KB

english, 2012

571

353. bInSiGHT: Bioinformatics Integration Sites Tool for Gene Therapy with High-Throughput Platforms

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

572

354. The Integration Profile of Lentiviral Vector in CD34+ Hematopoietic Stem Cells Is Dependent on the Cell Cycle Status of the Target Cell

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

573

355. Generation of a Barcoded Lentiviral Plasmid Library for Analyzing Clonal Complexity and Evaluation of Library Complexity Using Next Generation Sequencing

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

574

356. Codon Optimization of RD114-TR Envelope Glycoprotein Leads to Lack of Correct Processing and Therefore Correct Function of the Envelope

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

575

357. Regression of Schwannomas Induced by AAV-Mediated Delivery of Caspase-1

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

576

358. Directed Evolution of AAV for Enhanced Evasion of Human Neutralizing Antibodies

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

577

359. Novel, Shuffled, Human-Specific rAAV Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 240 KB

english, 2012

578

360. Enhanced Alpha-1 Antitrypsin (AAT) Expression and Decrease Immune Response with Isolated Limb Perfusion (ILP) Delivery of rAAV1-CB-hAAT

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

579

361. Arsenic Trioxide Treatment Increases the Transduction of Adeno-Associated Virus Serotype 2 (AAV2) Vectors Both In Vitro and In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

580

362. DNA Repair Dependent Viral Vectors Interrogate the Host DNA Damage Response and Offer Efficient Large Therapeutic Gene Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 299 KB

english, 2012

581

364. Biased Cardiac Gene Expression by Systemic Administration of AAV Vectors Irrespective of Mice Strain

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 640 KB

english, 2012

582

365. Characterization of Residual Plasmid DNA Impurities in Recombinant AAV; Correlation with Size of Transgene Expression Cassette

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 527 KB

english, 2012

583

366. Selection of an Optimal AAV Serotype for Gene Therapy in ALS

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 553 KB

english, 2012

584

367. High-Efficiency Transgene Expression in Monocyte-Derived Dendritic (moDCs) Mediated by Recombinant Adeno-Associated Virus Vectors Containing Short Subsets of Human CD11c Promoter

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 222 KB

english, 2012

585

368. High-Efficiency Transduction of Primary Human Monocyte-Derived Dendritic Cells by Recombinant Adeno-Associated Virus 2 Vectors Containing Mutations in Surface-Exposed Serine and Threonine Residues

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 236 KB

english, 2012

586

369. Two Complementary Methods To Identify AAVS1 Integrants

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

587

370. Optimization of Kidney-Targeted Gene Delivery for Cystinosis Using AAV

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

588

372. Targeted Mutagenesis of Ubiquitin-Binding Lysine Residues on the Adeno-Associated Virus (AAV)2 Capsid Improves Its Transduction Efficiency

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 299 KB

english, 2012

589

373. Sustained and Safe Inhibition of Hepatitis B Virus Replication In Vivo Using Helper-Dependent Adenovirus Vectors To Deliver Antiviral RNAi Expression Cassettes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 296 KB

english, 2012

590

374. Scavenger Receptor A (SR-A) and Scavenger Receptor Expressed on Endothelial Cells I (SREC-I) Are Receptors of Helper-Dependent Adenoviral Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 313 KB

english, 2012

591

375. Incorporation of Pseudomonas aeruginosa Epitope in Novel Adenovirus Fiber Loops To Circumvent Anti-Adenovirus Immunity and To Induce Protective Immunity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 182 KB

english, 2012

592

377. CD46 Does Not Affect the In Vivo Biodistribution of Species D Adenoviruses, Ad26 or Ad48 in CD46 Transgenic Mice Following IV Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 395 KB

english, 2012

593

378. Development of a Recombinase-Based Hybrid Baculoviral Vector for Sustained Transgene Expression

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 395 KB

english, 2012

594

379. Baculovirus Administration Triggered Adaptive Immune Response and Hindered the Subsequent Transgene Expression

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

595

380. Development of a Novel Targeted Adenovirus Vector Displaying Monobody Binding to Anti-Vascular Endothelial Growth Factor Receptor 2 in a Fiber Protein

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

596

381. Development of a Novel Adenovirus Vector Carrying microRNA-Targeted Sequences in the 3'-Untranslated Region of the pIX, E2A, or E4 Gene for Suppression of the Leaky Expression of Adenovirus Genes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

597

382. Studies on the Biodistribution Evaluation of Gene Therapy Products Using the Real Time-PCR

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

598

383. Tissue Specific piggyBac Gene Therapy Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

599

384. Quantification of Mature shRNA from Rat Tissues in a Biodistribution Study of pbi-shRNA™ STMN1 Lipoplex

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 342 KB

english, 2012

600

385. Efficient Enrichment of Cells with Programmable Nuclease-Induced Mutations by Surrogate Reporter

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 314 KB

english, 2012

601

389. Helper Independent Self-Inactivating Chimeric piggyBac Transposases for Genomic Targeting in Human Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 377 KB

english, 2012

602

392. A Simple, Flexible System for the Assay of Zinc Finger Nuclease Specificity and Activity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 241 KB

english, 2012

603

393. Application of Mammalian Artificial Chromosomes Suitable for Bioengineering of the Blood Brain Barrier and Delivery of Gene-Based Therapeutics

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 298 KB

english, 2012

604

394. A Novel Binary Transfection System for the Targeted Gene Delivery in Prostate Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

605

396. Gene Expression from Linearized Gene Expression Cassettes Capped with Multi-Arm DNA Junctions

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

606

397. Successful Gene Delivery with Polyamidoamine Dendrimers Targeted to the α Vβ3 Receptor Is Dependent on Caveolar Trafficking

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

607

398. Recent Advances Using Small Molecule Targeting for Bilamellar Invaginated Vesicle (BIV) Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 322 KB

english, 2012

608

399. Homogeneous Reducible Polypeptides for Nucleic Acid Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 266 KB

english, 2012

609

400. Vaccines Based on Disrupted Ad5 Against Small Molecule Drugs Are Efficacious in the Presence of Preexisting Ad5 Immunity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 281 KB

english, 2012

610

401. Combination of Doxorubicin and EGFR-Directed Immunonanoplexes Containing Vimentin/JAK3 siRNA for Anticancer Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

611

402. A Controlled DNA Complex Release System Comprising Injectable Auto-Forming Alginate Gel Including Amorphous Calcium Phosphate

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

612

403. Development of Dexamethasone Conjugated Low Molecular Weight Polyethylenimine as a Gene Delivery Carrier for Gene Therapy of Glioblastoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 260 KB

english, 2012

613

405. Pendant Polymer:Amino-β-Cyclodextin Guest:Host Complexes as Safe and Efficient Vectors for Nucleic Acid Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 246 KB

english, 2012

614

406. Efficient and Specific Co-Delivery of Vimentin siRNA and Doxorubicin with Aptamosomes for Combination Cancer Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 246 KB

english, 2012

615

407. Physical and Transfection Properties of Non-Viral Gemini Surfactant-Phospholipid Nanoparticles

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

616

408. Bioreducible and Acid-Labile Poly(Amido Amine)s for Efficient Gene Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

617

409. Arsonium-Based Lipophosphoramides: Antibacterial Effect and Transfection Ability — Both Activities in One Pot

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 412 KB

english, 2012

618

410. pDMAEMA-Based Polyplexes as Vectors for Retinal Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 350 KB

english, 2012

619

412. Novel Nanoparticles To Encapsulate microRNA for the Treatment of Stroke

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 479 KB

english, 2012

620

413. Hepatic Lentiviral Gene Transfer Is Associated with Mild Clonal Selection, but Not with Clonal Dominance or Tumour Formation in a Serial Transplantation Mouse Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 288 KB

english, 2012

621

414. Mobilization of Hematopoietic Stem Cells with Plerixafor or Plerixafor+G-CSF for Human Gene Therapy of Thalassemia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 312 KB

english, 2012

622

415. Development of a Lentiviral Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency (ADA-SCID)

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

623

417. Non-Invasive Monitoring of Insulin-Producing Cells In Vivo with Viral Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

624

418. Stable New Insulated Lentivectors towards Gene Therapy in Fanconi's Anemia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

625

420. Clinical Grade Lentiviral Vector Production for the Inherited Skin Disease, Netherton Syndrome

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

626

421. Engineered Insulin Secretion in Human Primary Thyroid Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

627

422. Compromised Platelet Fragmentation, Size, and Function Resulting from May-Hegglin Mutations of NMM-II

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 387 KB

english, 2012

628

423. TSLP shRNA Attenuates TGF-β1-Induced Renal Interstitial Fibrosis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 371 KB

english, 2012

629

424. Improving Axon Growth of Transected Spinal Cord Using Multichannel Collagen Conduits Carrying NT-3 Gene

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 400 KB

english, 2012

630

425. The Aurora A Kinase Inhibitor MLN8237 Significantly Enhances the Antitumor Activity of Oncolytic Measles Virus Derivatives in the Treatment of Glioblastoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 188 KB

english, 2012

631

426. Placental Growth Factor-2 Gene Transfer by Electroporation Improves Diabetic Sensory Neuropathy in Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 188 KB

english, 2012

632

427. Antisense Oligonucleotide-Mediated Exon Skipping Restores Primary Cilia Assembly in Fibroblasts Harbouring the Common Leber Congenital Amaurosis CEP290 Mutation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 231 KB

english, 2012

633

428. The Development of Non-Viral Vectors for Choroideremia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

634

429. Adenoviral and Lentiviral Vectors for Efficient Gene Transfer to Mouse Retina

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

635

430. CEP290 Minigene Model of Common Splice Site Mutation in Leber Congenital Amaurosis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 227 KB

english, 2012

636

431. CCR5-Transduced Neural Stem Cells Confer Accelerated and Enhanced Therapeutic Effect on Experimental Autoimmune Encephalomyelitis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

637

432. Intraocular Gene Therapy with Erythropoietin: Identification of Therapeutic Doses

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

638

433. A Highly Effective Equine Infectious Anaemia Virus-Based Lentiviral Gene Therapy Platform for the Treatment of Ocular Diseases

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 238 KB

english, 2012

639

434. AAV8-Mediated Gene Transfer to Cone and Rod Photoreceptors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 222 KB

english, 2012

640

435. Efficient Transduction of Tyrosine-to-Phenylalanine Mutated AAV2 Vectors Carrying Human ND4 Gene and Biodistribution Following Intravitreal Delivery in a Rodent Model — A Gene Therapy for Leber Hereditary Optic Neuropathy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 222 KB

english, 2012

641

436. A Recombinant Frataxin Protein Fused with a Cell Penetrating Peptide Prevents the Death of Fibroblasts with Knock-Out Frataxin Genes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 250 KB

english, 2012

642

437. The Brain Specific microRNA Regulates Neuronal Injury in Rat Intracerebral Hemorrhage Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

643

438. Biomaterial-Mediated Gene Delivery for Tissue Engineering of Articular Cartilage

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

644

439. Cartilage Matrix Recovery after Chondrocyte Transduction with a Dual Expressing Long Hairpin Plasmid Targeting IL-1 beta and TNF-alpha Knockdown

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 382 KB

english, 2012

645

440. Pathogenesis of Lumbar Spine Disease in Mucopolysaccaridosis and Response to Neonatal Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 353 KB

english, 2012

646

441. AAV Mediated RNAi and Antisense Knockdown of Intranuclear DMPK Transcripts in DM1 Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 378 KB

english, 2012

647

442. An Approach for Systemic Delivery of Embryonic Stem Cells into Mouse Skeletal Muscle

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

648

444. Efficient Myoblast Expansion for Urinary Incontinence Treatment

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 226 KB

english, 2012

649

445. Modulating Myoblast Differentiation for Improved Engraftment Potential

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

650

446. Blunted Ventilatory Response to Central Nervous Stimulation in a Canine Model of X-Linked Myotubular Myopathy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

651

447. Baculovirus as a Vector for Genetic Modification of Adipose-Derived Mesenchymal Stem Cells and Its Applications in Cartilage Tissue Engineering

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 238 KB

english, 2012

652

448. TGF-b1 Transduced Human Chondrocytes (TG-C) Formulated in Fibrin Glue Regenerated Cartilage in the Pig Cartilage Defect Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 215 KB

english, 2012

653

449. Recombinase Strategies To Make and Modify iPS Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 215 KB

english, 2012

654

450. Clinical Methods of Intramuscular Diaphragm Injections for Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 411 KB

english, 2012

655

451. Adenovirus Vaccine Vectors Encoding Full-Length Merozoite Surface Protein-1 from Plasmodium falciparum Effectively Induce Adaptive Immune Responses in Different Prime-Boost Combinations with Recombinant Protein

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 366 KB

english, 2012

656

452. Immunogenicity of an Engineered HIV-1 Clade a Consensus-Based Envelope DNA Vaccine

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 382 KB

english, 2012

657

453. Development of Intranasal Formulations for a Human Adenovirus Serotype 5-Based Vaccine with Potential To Bypass Pre-Existing Immunity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

658

454. Pushing the Limits of Expression: Highly Optimized Next Generation Molecular Adjuvants for Mucosa-Targeting DNA Vaccines

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

659

455. Enhanced DNA Vaccine with Protein Boost Delivered by EP Expands B and T Cell Responses and Neutralizing Phenotype In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 229 KB

english, 2012

660

456. Anti-HIV-1 Gene Therapy: In Vitro Safety and Efficacy of Human CD4+ T Cells Modified with a Gamma-Retrovirus Vector Conditionally Expressing the Bacterial Endoribonuclease MazF

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

661

457. P140K-Mediated In Vivo Selection Strategies Combining AMD3100 with O6-Benzylguanine and BCNU Significantly Enhances Selection of Gene-Modified T Lymphocytes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 218 KB

english, 2012

662

458. A Single Oral Administration of Human Adenovirus 40 Vaccine Against Allergy and Anaphylaxis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

663

459. Acute Intrahepatic CD8 T Cell Activation by Plasmid DNA Induces Defective CD8 T Cell Immune Response

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

664

460. A Solid Dosage Form for Buccal Delivery of Bacteria-Based Vaccines: Role of Excipients, Crystallinity and Drying Surface

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 242 KB

english, 2012

665

461. A Comprehensive Road Map for Risk Assessment of Sleeping Beauty Transposition Events in T-Cell Immunotherapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 228 KB

english, 2012

666

462. Evaluation of Immune Response to Treatment with TAG or FANG™ Vaccine by Assessing IFNγ Expression Using ELISPOT (Enzyme Linked Immunospot) Assay

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 228 KB

english, 2012

667

463. Elimination of Metastatic Tumors Using Gold Nanoshell-Enabeled Photothermal Therapy Pretreatment Followed by Adoptive T Cell Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 233 KB

english, 2012

668

465. Dual Transgenesis of T Cells with a CD44v6 CAR and a Suicide Gene for the Safe Eradication of Myeloid Leukemia and Myeloma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 249 KB

english, 2012

669

466. Attenuated CTLA-4 Inhibition in CD19-Targeted T Cells Expressing a Second-Generation CD28-Based Chimeric Antigen Receptor

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

670

468. Role of STAT3 Signaling in Dendritic Cell Based Immunotherapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

671

469. Uncovering T Cell Receptor Diversity by TCR nrLAM-PCR Sequencing

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

672

470. AdCD40L— From Model Systems to Clinical Trials for Malignant Melanoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

673

471. AdCD40L Immunostimulating Gene Therapy as a Sensitizer to T Cell Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

674

474. Lanatoside C Enhances the Therapeutic Efficacy of Secreted TRAIL Expressed by Human Neural Stem Cells in an Orthotopic Invasive Primary Glioblastoma Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

675

475. CD19-Specific T-Cell Therapy for Refractory Philadelphia-Chromosome-Positive Acute Lymphoblastic Leukemia Using PiggyBac Transposon-Based Gene Modification and Low Autoserum-Containing, but Xeno-Free and Tumor Cell Line-Free Culture System

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 232 KB

english, 2012

676

476. Targeting Gene Transfer in Pancreatic Adenocarcinoma with Cell Surface Antigens

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

677

477. Multipotent VΔ2-Negative γΔT-Cells after CMV-Reactivation in Allogeneic Stem Cell Transplantation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

678

478. Inhibition of Metastasis from Targeted Interleukin 12 Immunotherapy and Revelation of the Mechanism by Cell Profiling

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

679

479. Adenoviruses-Mediated Up-Regulation of p53 Expression Produces Cytotoxic Effects on INK4A/ARF-Defective Mesothelioma and Increases the Susceptibility to Chemotherapeutic Agents and Small G Protein Inhibitors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

680

481. Effects of Angiotensin Type 2 Receptor Overexpression on Gene Expression Profiles in Human Prostate Cancer Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 209 KB

english, 2012

681

482. Inhibition of Breast Tumor Growth by Adenoviral-Mediated p16 Gene Transfer Via Combined Effects of Cell Cycle Arrest, Senescence and Apoptotic Induction, and Angiogenesis Inhibition

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

682

483. Production and Evaluation of Lenti-hWASP-Vectors for Wiscott Aldrich Syndrome

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 185 KB

english, 2012

683

484. Gene Therapy for Wiskott Aldrich Syndrome: Sustained Expression from the MND Promoter in Platelets and Blood Cells upon Serial Transplantation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

684

485. Illumina Platform Sequencing Provides Adequate Read Lengths To Map Lentiviral Vector Insertion Sites in Human Cells and Uncovers Both Mis-Mapped and False Positive Reads from Traditional LAM-PCR and nrLAM-PCR Products

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

685

486. Use of the In Vitro Immortalization Assay To Quantify the Impact of Vector Design and Integration Pattern on Insertional Mutagenesis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 205 KB

english, 2012

686

487. Large-Scale Expansion of Functional Regulatory T Cells Using a Gas-Permeable Rapid Expansion Cultureware (G-Rex)

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 205 KB

english, 2012

687

488. Lentiviral Gene Therapy for X-SCID Using HSCs Mobilized by Treatment with G-CSF and AMD 3100

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 233 KB

english, 2012

688

489. Humoral Tolerance to FVIII in Hemophilia A Mice Using Cytokine-Conditioned Dendritic Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 197 KB

english, 2012

689

490. Abstract Withdrawn

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 191 KB

english, 2012

690

491. AAV Delivery to the Nasopharynx for Pharmacologically-Regulated Systemic Expression of Therapeutic Proteins

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 191 KB

english, 2012

691

493. Comparison of Safety and Effectiveness between Gamma-Retroviral, Lentiviral and Foamy Virus Gene Transfer Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

692

494. A Hematopoietic Stem Cell Expansion Medium Increased Transduction Efficiency for Human CD34+ Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 430 KB

english, 2012

693

495. Validation of Cellular Function and Imaging Capability of CD34+ Stem Cells Labeled with a 19F MRI Contrast Agent

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 406 KB

english, 2012

694

496. Optimised Factor VIII Vectors for Gene Therapy of Haemophilia A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 406 KB

english, 2012

695

497. Human Erythropoietin Gene Delivery Using an Arginine-Grafted Bioreducible Polymer System

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 442 KB

english, 2012

696

498. Development of In Vivo Gene Therapy of Hemophilia A Mice by Intra-Bone Marrow Injection of Lentiviral Vectors Containing a B-Domain Variant of Human Factor VIII

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 205 KB

english, 2012

697

499. Upregulation of the Pluripotency-Associated miRNA 302-367 Cluster Using Engineered Transcription Activator-Like Effector (TALE) Activators

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 205 KB

english, 2012

698

500. Light-Inducible Spatiotemporal Gene Regulation Using Engineered Transcription Factors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 229 KB

english, 2012

699

502. Differential Regulation of Multiple Transgenes in AAV Vectors by Alternative Splicing

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

700

503. Transcription of MLV Proviruses Involves RNA Polymerase II-Associated Looping of the 5’ and 3’ LTRs

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

701

507. Transcriptional Repression of the Rhodopsin Gene Via Zinc-Finger Technology for the Treatment of the Dominant Retinal Degenerations

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

702

508. Synthetic Promoter Design of Cell Type-Specific Adeno-Associated Viral Vectors in the Retina

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

703

510. Circulating microRNA as a Potential Biomarker of Ischemic Stroke

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 230 KB

english, 2012

704

512. Comparison of Commercially Available Furin ELISA Kits To Assess Furin Knockdown in cGMP Manufactured FANG™ Vaccines

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 218 KB

english, 2012

705

513. The Immunosuppressant Drug Rapamycin Relieves Lentiviral Transduction Resistance in Human Hematopoietic Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 218 KB

english, 2012

706

514. FVIII Expression and Secretion in Circulating Blood Cell Types Capable of Correcting Bleeding in Hemophilia A Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 234 KB

english, 2012

707

516. Protein Kinase C Activators Enhance Zinc Finger Nuclease-Driven Genome Editing in Hematopoietic Stem/Progenitor Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 199 KB

english, 2012

708

517. CD15-Enrichment of Donor Primary Murine Neural Stem Cells Increases Total Engraftment and Results in Wider Dissemination within Host Brains

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

709

518. Radiation and Temozolomide Chemotherapy Do Not Inhibit the Oncolytic Adenovirus Loaded Carrier Cells to Effectively Deliver Their Payload

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

710

519. High-Level Ectopic Expression of CFTR Imposes a Selective Disadvantage to Airway Epithelial Stem/Progenitor Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 200 KB

english, 2012

711

521. Determinants of Extrahepatic Biliary-Derived Cell Transdifferentiation into Endocrine beta-Like Cells In Vitro

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

712

522. Isolation and Culture of Neuronal Stem Cells Which Directed into Purkinje Cells To Be Used for Brain Damage and Epilepsy Treatment in Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

713

523. Vector Genome Length and Self-Complementarity Affect AAV Capsid Uncoating

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

714

524. Patterns of scAAV Vector Genome Insertion Associated with Oncogenic Events in a Mouse Model for Genotoxicity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 187 KB

english, 2012

715

525. Absence of Integration Hotspots in Non-Human Primates after Intravenous Injection of AAV2/5-AAT-coPBGD

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

716

526. Intravasuclar Delivery of RAAVRH.8 Generates Widespreading Transduction of Neuronal and Glial Cell Types in the Adult Mouse Central Nervous System

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

717

527. Altering Tissue Glycan Patterns Improves the Transduction Profile of AAV9 Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

718

528. rAAV8/9-Mediated Muscle Transduction with Tacrolimus in Non-Human Primate

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

719

529. Evaluation of Porcine AAVs as Vaccine Vectors Against Avian Flu and Zaire Ebolavirus Infections

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

720

531. TCR Gene Editing Results in Effective Immunotherapy of Leukemia without the Development of GvHD

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 235 KB

english, 2012

721

532. Donor T Cells Specific for Multiple Leukemia Antigens for Adoptive Immunotherapy of Myeloid Leukemias after Stem Cell Transplantation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 235 KB

english, 2012

722

534. TGF-β as a Modifier of Response to Adoptive Cell Therapy for Melanoma: Enhanced Tumor Treatment in Animal Models and a Highly Significant Correlation between Clinical Response and TGF-β Genotypes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

723

535. T Cells Redirected Against HER2 for Adoptive Immunotherapy for HER2-Positive Osteosarcoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 242 KB

english, 2012

724

536. Infusion of CD19-Directed/Multivirus-Specific CTLs Post HSCT for B Cell Malignancies

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 222 KB

english, 2012

725

537. The 2nd Generation “siTCR” Vector: Improvement of Efficacy and Safety in TCR Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 237 KB

english, 2012

726

538. A Novel Immune Receptor Expressed by T Cells for Universal Targeting of Diverse and Multiple Tumor Associated Antigens

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

727

539. Selective Targeting and Disruption of Tumor-Associated Vasculature in Humans with an Engineered Oncolytic Poxvirus

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

728

541. Improving the Efficacy of Adoptive T Cell Therapy by Combination with Viro-/Immuno-/ Radio-Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

729

542. Efficient HSV-1 Retargeting by Modification of Glycoproteins D and B

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

730

543. Phase 1 Study of CG0070, a GM-CSF Expressing Oncolytic Adenovirus, in Non-Muscle Invasive Bladder Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 175 KB

english, 2012

731

544. Kinetic Analysis of Oncolytic Virus Spread through Large Lymphoid Tumors after Systemic Administration

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 175 KB

english, 2012

732

545. Transductionally-Retargeted Infectivity Selective Oncolytic Adenovirus with Redesigned AB-Loop Targeting Cell Surface Mesothelin

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 210 KB

english, 2012

733

546. Mesenchymal Stromal Cells Are Promising Candidates as Delivery Vehicles for Systemic Oncolytic Measles Virotherapy in Models of Acute Lymphoblastic Leukaemia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

734

547. Lentiviral Vector Transduced CD34+ Cells for the Treatment of Wiskott-Aldrich Syndrome

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 209 KB

english, 2012

735

548. Liver Gene Therapy by Lentiviral Vectors Allows Partial Correction of Two Hemophilia B Dogs and Revert Pre-Existing Anti-Transgene Immunity in Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

736

549. High-Level Clustering of Vector Integrations in the German WAS Clinical Gene Therapy Trial

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

737

550. Transplantation of Autologous MGMTP140K Gene-Modified CD34+ Cells in Glioblastoma Patients Allows for Increased Chemotherapy Administration and Extended Survival

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

738

551. Broadening the Donor Pool by Disrupting HLA Expression in Hematopoietic Stem Cells without Compromising Stem Cell Properties

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

739

552. Gene Therapy of Human Blnk Deficiency in NOD/SCID/gc ko Murine Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 237 KB

english, 2012

740

553. Integration Site Analysis Indicates Decade-Long Safety of Gammaretroviral-Modified Chimeric Antigen Receptor T-Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 213 KB

english, 2012

741

556. Pharmacological Modulation of Humoral Immunity in a Non-Human Primate Model of AAV Gene Transfer for Hemophilia B

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

742

557. Characterization of AAV T Cell Epitopes Presented by Splenocytes from Normal Human Donors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

743

559. Phase 1 Study of HSVTK-CD34 Modified T Cells Therapy Following Haematopoietic Stem Cell Transplantation (HSCT) without Serotherapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

744

560. Autophagy Induced by Oncolytic Adenoviruses Combined with Temozolomide Triggers Antitumor Immune Responses Preclinically and in Cancer Patients

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

745

561. Expansion of FoxP3+ Regulatory T Cells in Mucopolysaccharidosis Type I Mice Prior to Lentiviral Gene Transfer Promotes Tolerance to α-l-Iduronidase

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

746

562. Interactions of Adenovirus Vectors with Coagulation Factor X and Natural Antibodies: Lack of an Absolute Requirement for Factor X in Liver Transduction by Adenovirus Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 275 KB

english, 2012

747

563. miR-126 Controls Hematopoietic Stem Cell Proliferation by Regulating PI3K/AKT Signaling

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 275 KB

english, 2012

748

565. Mechanisms of Stem Cell Therapy in Cystinosis Treatment

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

749

566. Extreme Skewing of X-Chromosome Inactivation in Female Carriers of X-CGD Can Be Reversed during iPSC Reprogramming To Restore Neutrophil Function

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

750

567. Identifying Molecular Targets for the Rejuvenation of Aged Muscle Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

751

568. Zinc-Finger Nuclease Mediated Correction of α-Thalassemia in iPS

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 210 KB

english, 2012

752

569. Correction of Artemis Deficiency in Murine Hematopoietic Stem Cell by I-Sce1 Meganuclease and Artemis Recombination Matrix Mediated Homologous Recombination

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

753

570. Efficient Generation of Induced Pluripotent Stem Cells by the Inhibition of PTEN Tumor Suppressor

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

754

571. Manufacture of Clinical-Grade Lentiviral Vectors for Ex Vivo Use

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

755

572. Efficient High Titer Production of Recombinant Lentiviral Vectors for Cancer Immunotherapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

756

574. Magnetic Colocalization of Viral Vectors and Target Cells Improves Transduction Efficiency in Human Hematopoietic Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

757

575. Optimizing the Manufacture of CAR-T Cells for Clinical Applications

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

758

576. Exploiting the Mechanism of Intron-Splicing in Insect Cells To Produce Viral Vectors Harboring Toxin Genes for Cancer Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

759

577. Ratios of Full to Empty Virions of AAV8 Vector Preparations Negatively Impact on Transduction Efficiency and Liver Toxicity in the Liver-Directed Gene Transfer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

760

578. Large-Scale Production of a Maraba-Based Oncolytic Virus

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 201 KB

english, 2012

761

579. Complete Tropism Restriction by Display of High Affinity Ligands on Adeno-Associated Viral Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

762

580. Insight into the Mechanism of Increased Transduction of AAV Vectors with Ad Proteins E1B55k/E4orf6 and Generalization to Other Substrates of the Cellular DNA Damage Response Pathway

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

763

581. Toxicology and Liver Transduction Efficacy Evaluation of a Recombinant Adeno-Associated Viral Vector in Non Human Primates as a Potential Treatment for Acute Intermittent Porphyria

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 198 KB

english, 2012

764

582. Expression of Therapeutic Levels of Human Clotting Factor IX in Non Human Primates from an AAV5-Based Gene Therapeutic Vector Generated Using a Fully-Scalable GMP-Compliant Production System

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

765

583. Integration Site Analysis Following Transduction with the Novel Recombinant AAVHSC15 Reveals Trends in Proviral-Chromosome Junction Formation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

766

584. Optimization of rAAV for Anti-Glioma Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 227 KB

english, 2012

767

585. Strategy for rAAV-Mediated Transduction of Common Marmoset Skeletal Muscle To Generate NHP DMD Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

768

586. Distinct Transduction Difference between Adeno-Associated Virus Type 1 and Type 6 Vectors in Human Polarized Airway Epithelia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 218 KB

english, 2012

769

587. Transduction and Expression in Brain after Systemic Delivery of Single Stranded AAV9 in Adult Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 376 KB

english, 2012

770

589. Enhanced Fracture Healing Utilizing In Vivo Biopanning of Adeno-Associated Virus from a Directed Evolution Library

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 376 KB

english, 2012

771

590. Primary Human Keratinocyte-Selective AAV Targeting Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

772

591. Directed Evolution To Create Choroid Plexus-Permissive AAV Capsid Variants: Toward Novel Clinical Management of Lysosomal Storage Disorders

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 175 KB

english, 2012

773

593. High-Efficieny Transduction of Primary Human Hematopoietic Stem Cells with Recombinant Adeno-Associated Virus Vectors at a Low Multiplicity of Infection

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

774

595. Artificial RNA Switches for Regulation of Gene Transfer by Adenovectors and Oncolytic Adenoviruses

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

775

597. Systemic Delivery of Chitosan-PEG-FA-Conjuatged Oncolytic Adenovirus Nanocomplexes for Folate Receptor-Overexpressing Cancer Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

776

598. Abstract Withdrawn.

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

777

599. Evaluation of the Cytokines SCF and IL-3 and Charged-Based Polymers for Targeting of Adenovirus Vectors to Human Hematopoietic Progenitor Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

778

600. Local Sustained Delivery of Oncolytic Adenovirus with Injectable Alginate Gel for Cancer Virotherapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

779

601. Linearized Oncolytic Adenoviral Plasmid DNA Delivered by Bioreducible Polymers

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

780

602. A Novel sLRP6E1E2 Inhibits Canonical Wnt Signaling, Epithelial-to-Mesenchymal Transition, and Induces Mitochondria-Dependent Apoptosis in Lung Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

781

603. Generating a Replication Competent Adenovirus Vector That Targets the c-Met Receptor

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 302 KB

english, 2012

782

604. Oncolytic Herpes Simplex Virus Shows Synergistic Effects with Rapamycin Against Triple-Negative Breast Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 287 KB

english, 2012

783

605. Magnetic Nanoparticle Uptake and Gene Delivery for Magnetically Guided Endothelial Cell Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 429 KB

english, 2012

784

606. Chimpanzee Adenovirus Expressing microRNA as a New Therapeutic Strategy Against HPV-Related Cervical Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 302 KB

english, 2012

785

607. Targeting 2A-Fusions to Endogenous Genes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 301 KB

english, 2012

786

608. Optimisation of the ‘FMDV’ 2A Co-Expression System for Gene Therapies

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 325 KB

english, 2012

787

609. Preclinical Evaluation of Bifunctional Short Hairpin RNAs (shRNA) Against Steroid Receptor Coactivator-3/Amplified in Breast Cancer 1 (SRC-3) and Steroid Receptor Coactivator-1 (SRC-1) in Breast Cancers

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

788

610. Abstract Withdrawn

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 182 KB

english, 2012

789

611. Genetic Modification of Cancer Cells Using Non-Viral, Episomal S/MAR Vectors for In Vivo Tumour Modelling

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

790

612. Development of Non-Viral Vectors for the Prophylactic Gene Therapy of Birt—Hogg—Dubé (BHD) Syndrome

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 225 KB

english, 2012

791

613. Closed-Ended Linear Duplex AAV (celdAAV) DNA for Non-Viral Gene Transfer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

792

614. A Novel Viral Promoter Exhibits Bi-Directional and Highly Inducible Gene Expression Following Stable Delivery in Mammalian Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

793

615. Efficiency of AAV Versus DNA Nanoparticle Formulation in Retinal Gene Targeting

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 231 KB

english, 2012

794

616. A Novel Plasmid-Based Therapy Using a Bi-Functional shRNA Vector Targeted to Mesothelin Reduces Pancreatic Cancer Cell Aggressiveness

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 231 KB

english, 2012

795

617. Broadening the DNA-Recognition Potential of Homeodomains To Create New Modules for Targeted Genome Editing

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

796

618. Therapeutic Gene Delivery of Facor IX Mediated by Ultrasound and Microbubbles in Murine Models

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

797

619. IL30 — A Novel Cytokine Candidate for Prevention and Treatment of Biological Therapy-Induced Liver Injury and Hepatitis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

798

620. Electroporation Enhanced Human Kallistatin Expression for the Prevention of TNBS-Induced Colitis in Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

799

621. Gene Delivery to Skin by Electroporation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

800

622. Intranasal Delivery of HMGB1 Inhibitory Heptamer Peptides Confers Robust Neuroprotection in the Postischemic Brain

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

801

623. Intravenous PMO Delivery System with Bubble Liposomes and Ultrasound Exposure into Skeletal Muscles of the mdx Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 220 KB

english, 2012

802

624. Long Term Transgene Expression in Rat Liver Using a Novel Electromotor-Driven Hydrodynamic Gene Injector

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

803

625. Differential Perturbations of the Salivary Gland Proteome Following AAV-Mediated or Ultrasound-Assisted Gene Transfer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

804

626. Transfection of ‘Hard To Transfect’ Cells Using Electrospray: ‘Sprayfection’

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 357 KB

english, 2012

805

628. Efficient Hydrodynamic Limb Perfusion of an AAV9 Mini-Dystrophin in Adult GRMD Dogs by a Clinically Used, Pressure- and Speed-Controlled Pump

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

806

629. Refined Ultrasound-Mediated Gene Delivery Using Customized Microbubbles In Vitro

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

807

630. Optimal Electrogene Transfer Method for Human Mesenchymal Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 229 KB

english, 2012

808

634. Fusogenic Cell Entry of Bilamellar Invaginated Vesicle (BIV) Complexes with or without Small Molecules for Targeted Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 218 KB

english, 2012

809

635. Development of Plasmid-Lipoparticle Systemic Delivery System That Overcome the Paradox of PEG-Stabilization While Maintaining High Transfection Potency

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 218 KB

english, 2012

810

636. Development of Non-Viral Gene Delivery Particles Based on the Dynein Light Chain Rp3

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 377 KB

english, 2012

811

638. Combinational Anti-Cancer Therapy with Doxorubicin and EGFR-Targeted Immunonanoparicles Containing IL12/Salmosin Genes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 391 KB

english, 2012

812

639. Development of Novel Phosphonium-Containing Block Copolymers for siRNA Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 181 KB

english, 2012

813

640. Aptamer Mediated Delivery of Synthetically Derived Modified mRNA

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 394 KB

english, 2012

814

642. Co-Delivery of Minicircle-Endostatin and ZD6474, a Small Molecule Tyrosine Kinase Inhibitor from Biodegradable Cationic Micelles for Cancer Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 381 KB

english, 2012

815

643. Development of Anionic Nanocomplexes for Targeted SiRNA and Plasmid DNA Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

816

645. Phosphonium-Containing Macromolecules for Nonviral Gene Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 338 KB

english, 2012

817

646. Development of pH Responsive Cationic Amphipathic Peptides for the Delivery of Plasmid DNA and siRN A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 324 KB

english, 2012

818

647. Stabilized Quaternary Nanoparticles for Targeted Delivery of siRNA

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 640 KB

english, 2012

819

648. DMSO Shock Yields Highly Efficient Transient Transfection by PEI/DNA Nanoparticles

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 476 KB

english, 2012

820

651. A Novel iPSC-Based Strategy To Correct the Bleeding Phenotype in Hemophilia A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

821

652. Long-Term Follow-Up of Hemophilia A Dogs for 2-7 Years Demonstrates Sustained Factor VIII Expression after AAV-Mediated Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

822

653. Proliferative Endothelial Progenitors as a Therapeutic Vehicle for the Treatment of Hemophilia

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

823

654. Early AAV-Mediated Intrauterine Gene Therapy at 0.4G in Non-Human Primates

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

824

655. Dose-Response Relationship of Factor IX Expression in Non-Human Primates Following IV Administration of AAV8 Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

825

656. Optimisation and Validation of Protocols for Vector Detection in Circulation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

826

657. In-Mouse Propagation of Hemophilic Hepatocytes toward Gene Correction and Cell Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

827

659. A Study of Wnt and Notch Signaling in Muscle Stem Cells from Progeria Mouse Models

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 236 KB

english, 2012

828

660. Novel GNE Mutations in Patients with Autosomal Recessive Hereditary Inclusion Body Myopathy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

829

661. Development of a Novel DNA Vaccine Strategy for Prevention of Clostridium difficile Infection

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

830

662. Broad Neutralizing Immune Responses Against Hepatitis C Virus Induced by Vectored Measles Virus and a Recombinant Envelope Protein Boost

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

831

663. In Vitro Characterization of the Anti-Viral Activity of TT-034, a RNAi-Therapeutic Against the Hepatitis C Virus

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 182 KB

english, 2012

832

664. Electroporation-Mediated Genetic Vaccination for Antigen Mapping: Application to Plasmodium Falciparum VAR2CSA Protein

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 182 KB

english, 2012

833

665. Combinatorial Approach Using MGMT P140K Transgene and Multiplexed RNAs in Gene Therapy Against HIV-1

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 218 KB

english, 2012

834

666. Type-I IFN Signaling Is Required for the Induction of Antigen-Specific CD8+ T Cell Responses by Adenovirus Vector Vaccine in the Gut-Mucosal Compartment

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

835

668. Capsid Modified AAV2 Vectors Are Capable of Generating Vaccine-Mediated Protection

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 506 KB

english, 2012

836

669. Antibody-Mediated Neutralization and Enhancement of Measles Virus Infectivity in Microglial Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 479 KB

english, 2012

837

670. Modeling Pre-Existing Immunity to Adenovirus in Rodents: Immunological Requirements for Successful Development of an Ad5-Based Ebola Vaccine

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 526 KB

english, 2012

838

671. Immunological Response to CD40L Coding, Replicating and Non-Replicating Adenoviruses

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 215 KB

english, 2012

839

672. Adoptive Therapy Evaluating Human IL13-Zetakine T Cells Engineered To Target Glioblastomas Expressing IL13Rα2, a Biomarker Predicting Poor Prognosis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 232 KB

english, 2012

840

673. Myeloid Derived Suppressor Cells in Glioma Differentiate towards an Antigen Presenting Cell Phenotype upon Interleukin-12 Vector Based Immunotherapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

841

674. VSV-Expressed cDNA Libraries Cure Established Melanoma and Identify Repertoires of Antigens Which Co-Operate To Stimulate Protective Tumor Immunity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

842

676. Short-Hairpin RNA Interference with CD44v6 Reveals a Complex Role in Chemoresistance and Leukemogenicity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 232 KB

english, 2012

843

677. Evaluation of Anti-Tumor Effects Mediated by Engineered T Lymphocytes Expressing a CD19 Specific CAR for B Cell Lymphoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 232 KB

english, 2012

844

678. A Pre-Clinical Dataset Exploring the Effects of a 19F-MRI Cellular Label on Dendritic Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 288 KB

english, 2012

845

679. Preliminary Safety Results from the IMPACT Study — A Phase 2-3 Clinical Study in Patients with Advanced Colorectal Carcincoma with the Immunomodulator MGN1703

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 250 KB

english, 2012

846

681. Syngeneic Hematopoietic Stem Cell Transplantation Enhances the Antitumor Immunity of Intratumoral Type I Interferon Gene Transfer for Sarcoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 416 KB

english, 2012

847

682. A Protocol for Ex Vivo Expansion of Effector T Cells That Are Resistant to Oxidative and Apoptotic Stress

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 416 KB

english, 2012

848

684. Systemic Administration of Platelet Vector Containing Inactivated Sendai Virus Particle Eradicates Melanoma in Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 434 KB

english, 2012

849

685. A Transcriptionally Targeted, Systemically Administered AAV-sTRAIL in Combination with Lanatoside C for Glioblastoma Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 459 KB

english, 2012

850

686. Intercellular Transfer of microRNAs Via Tumor Microvesicles

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

851

688. Matrix Metalloproteinase Responsive, Proximity-Activated Targeting Polymeric Nanoparticles for siRNA Delivery to Tumor Metastases

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 707 KB

english, 2012

852

689. Modulating Cancer Progression with Zymogen Protein C in a Murine Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 751 KB

english, 2012

853

690. High Resolution In Vivo Bioluminescent Imaging for the Study of Bacterial Vector Trafficking

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 203 KB

english, 2012

854

691. Developing Inhibitor-Directed Enzyme Prodrug Therapy for the Treatment of Prostate Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 203 KB

english, 2012

855

693. Over-Expression of miR-145 Enhances the Effectiveness of HSVtk Gene Therapy for Malignant Glioma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 197 KB

english, 2012

856

695. Evaluation of a Novel Oncolytic Herpes Simplex Virus Type-1 Encoding Human Sodium Iodide Symporter for Human Prostate Cancer Radiovirotherapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 240 KB

english, 2012

857

696. Oncolytic Vaccinia Virus Infection of Tumor Vasculature in a Window-Chamber Tumor Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

858

697. Immunomodulatory Transgene Armed Oncolytic Measles Virus Induces Anti-Tumor Immunity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

859

698. The Role of Receptor Density in Influencing Cytolytic Activity of Retargeted VSV

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 181 KB

english, 2012

860

699. Abstract Withdrawn

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 181 KB

english, 2012

861

700. Adenoviral Vectors Retargeted towards Receptor Tyrosine Kinase EphA2 Show Increased Tumor to Liver Ratio after Systemic Application

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 205 KB

english, 2012

862

701. Polymorphonuclear Neutrophils(PMNs) Are Cytotoxic for Measles Virus (MV)-Infected Tumor Cells Ex-Vivo, but Their Role in MV-Mediated Oncolysis In-Vivo Is Tumor-Type Dependent

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

863

702. Improved Antitumor Effect of Adenovirus-Mediated Interferon Therapy in Combination with Chemoradiotherapy in a Syngeneic Immunocompetent Hamster Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

864

703. Serotype Chimeric Oncolytic Adenovirus Coding for Granulocyte-Macrophage Colony-Stimulating Factor (GMCSF) for Treatment of Soft Tissue Sarcoma in Rodents and Humans

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

865

705. Molecular and Immunological Complementation between Oncolytic Adeno- and Vaccinia Virus: Paving the Way for Next Generation Cancer Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

866

706. Oncolytic Vaccinia Virus Armed with GM-CSF: Studies on Safety, Efficacy, Anti-Viral and Anti-Tumor Immune Response in Immunocompetent and Immunodeficient Rodents

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

867

707. Oncolytic Echovirus 4 as a Potent Virotherapy Agent Against Human Esophageal Squamous Cell Carcinoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 238 KB

english, 2012

868

708. Oncolytic Vaccinia Viruses Deleted in the Gene Encoding the Small Subunit of Ribonucleotide Reductase for the Treatment of Bladder Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

869

709. Virological and Immunological Data in Cancer Patients Treated with a Fully Serotype 3 Oncolytic Adenovirus Ad3-hTERT-E1A

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

870

710. Evaluation of Membrane-Anchored Versions of Interleukin-12 as Transgenes in Armed Oncolytic Adenoviruses

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

871

711. Phase II 2:1 Randomized Adjuvant Study of the “Triad” Autologous (FANG™) Vaccine, Incorporating Bifunctional shRNAfurin and GMCSF Transgene Expression, in Stage IIIc Ovarian Epithelial Carcinoma: Preliminary Data

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

872

712. Conditionally Replicating Adenovirus Influences Intracranial Infiltration of Regulatory T Cells in Experimental Mouse Model of Brain Tumor

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

873

714. Novel Strategies To Modulate Pre-Existing Anti-Factor VIII Immune Responses in Hemophilia A Inhibitor Mice Treated with Factor VIII Plasmid-Mediated Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 320 KB

english, 2012

874

715. A Dynamic Shift from Cytokine to Cell Death-Mediated Stimuli Orchestrates Macrophage-Driven Local Neutrophilic Inflammation in Response to Adenovirus In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 301 KB

english, 2012

875

716. Transient Transgene Repression Post-Gene Delivery Improves Long-Term Expression In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 307 KB

english, 2012

876

717. Phase I Trial of Escalating Doses of SGT-53 Infusion in Combination with Taxotere in Advanced Solid Tumor Patients

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

877

718. Anti-Luciferase Immune Response after Sleeping Beauty-Mediated Gene Transfer In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

878

719. Fc gamma Receptor Polymorphisms as Prognostic and Predictive Biomarkers for Oncolytic Adenovirus Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 490 KB

english, 2012

879

720. Reducing Clearance of Lentiviral Vectors by Immune Cells in Order to Improve Safety and Efficacy of Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 444 KB

english, 2012

880

721. Non-Contact Helium Based Plasma for the Efficient Delivery of DNA Vaccines

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 649 KB

english, 2012

881

722. Targeting siRNA Toxicity to HER2+ Tumors: Multi-Pronged Attack and Implications for Drug-Resistance

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 364 KB

english, 2012

882

723. Multifunctional Lipid-Polymer Nanoparticles for Enhanced Serum Stability and Intracellular Delivery of Oligonucleotides

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 364 KB

english, 2012

883

724. Lung Tumor Metastasis Inhibition with Modified siRNA Targeting Cell Cycle

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

884

725. Induction of CD4+CD25+ T Cells and Control of Cardiac Allograft Rejection by CD40 RNAi in Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

885

726. Effect of 5’ Terminal Triphosphate in Exogenous siRNA and shRNA on Gene Silencing and Innate Immune Response

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

886

728. siRNA-miRNA Chimera Initiation Both siRNA-Induced and miRNA-Induced RNAi

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

887

729. ZIP4 Is a Novel Therapeutic Target in Pancreatic Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

888

730. Targeting Androgen Receptors in Hormone-Dependent and Castration Resistant Prostate Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 255 KB

english, 2012

889

731. Specific RNA Cleavage by Oligodeoxyribozyme Suppresses Proliferation of Drug-Resistant Leukemia Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

890

732. Effects of hEag1 shRNA Expression Vector on Cell Viability of Glioma Cells In Vitro

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

891

733. Dose-Dependent Knocking-Down on Eag1 from Glioma Cells in Culture Caused by Synthetic siRNAs

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 228 KB

english, 2012

892

734. nNOS shRNA Expression Vector in Combination with Interferon Gamma To Reduce the Viability of Human Glioma Cell Lines

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 203 KB

english, 2012

893

735. nNOS Short Hairpin RNA Expression Vector To Reduce the Glioma Cells Viability

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 203 KB

english, 2012

894

736. RNA Interference on nNOS in Combination with Neomycin To Reduce the Viability of Human Glioma Cell Lines

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 443 KB

english, 2012

895

737. Defective Proliferation and Myogenic Differentiation Potential of Muscle-Derived Stem Cells Isolated from Dystrophin/Utrophin Double Knock-Out Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 409 KB

english, 2012

896

738. Cyclooxygenase-2 Plays a Critical Role in BMP4GFP Transduced Muscle Derived Stem Cell Mediated Bone Healing in the Critical Sized Calvarial Defect Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 888 KB

english, 2012

897

739. Bioprinted Amniotic Fluid-Derived Stem Cells Accelerate Wound Healing in Skin

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 665 KB

english, 2012

898

740. Prevention of Radiation-Induced Lung Injury by Administration of Gene-Modified Mesenchymal Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.09 MB

english, 2012

899

741. In Vivo Real-Time Imaging of Cell Dynamics in Tissues of a Living Mouse by Confocal Microscope with the Suction Device

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 621 KB

english, 2012

900

742. Tissue Factor-Triggered Procoagulant Activity of Murine/Human Mesenchymal Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

901

745. Viral-Based Dual-Color Imaging for Cell Division and Fusion In Vitro

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 232 KB

english, 2012

902

746. A Red-Nucleus Retro-Vector for Rat Muscle-Derived Stem Cell Modification

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 257 KB

english, 2012

903

747. Generation of Vascularized Nervous or Insulin-Secreting Tissues

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

904

748. Chondrogenesis of Mesenchymal Stem Cells In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

905

749. Long-Term Cultured Human Term Placenta-Derived Mesenchymal Stem Cells (MSC) of Maternal Origin Displays Plasticity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

906

750. Spectral Karyotyping of Multipotent Stromal Cells during In Vitro Expansion

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 226 KB

english, 2012

907

751. Autologous Circulating Angiogenic Cells Treated with Osteopontin and Delivered Via a Collagen Scaffold Enhances Wound Healing in the Alloxan-Induced Diabetic Rabbit Ear Ulcer Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

908

752. Pharmacokinetic-Guided Delivery of Mesenchymal Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

909

753. A Simple and Effective Method To Generate Lentiviral Vectors for Ex Vivo Gene Delivery to Mature Human Peripheral Blood Lymphocytes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

910

754. A Simple and Scalable Method To Concentrate Measles Virus Glycoprotein-Pseudotyped Lentiviral Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 227 KB

english, 2012

911

755. Immobilized Colanic Acid Degrading Enzyme Reactor Chromatography and Its Application in SuperClean Therapeutic Plasmid DNA Production

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 213 KB

english, 2012

912

756. Absolute Determination of Single-Stranded and Self -Complementary AAV Vector Genome Titers by Digital PCR

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 213 KB

english, 2012

913

757. Gene Transfer to Human Hematopoietic Cells with GaLV Pseudotyped Retroviruses Concentrated from Serum-Free Supernatants by PEG Precipitation with Polyvalent Immunoglobulin

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 251 KB

english, 2012

914

758. A Novel Platform of Genetically-Modified Cell Transplantation Using 3D Spheroid Culture System on Micropatterned Substrates and Polyplex Nanomicelles

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

915

759. cGMP Plasmid Manufacturing and Release Criteria for Phase I/II Trials

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

916

760. Human CD20-Transduced Packaging Cell Lines Express hCD20Δ Alternative Transcripts but Produce Low Level of Retroviral Particles Carrying Spliced RNA

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 223 KB

english, 2012

917

761. Large-Scale cGMP Manufacture of a Plasmid Vector for Cystic Fibrosis Gene Therapy Clinical Trials

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

918

762. Characterisation of Viral Count by Nanoparticle Tracking and Analysis (NTA) Technique and Its Application to Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

919

763. Peak Supply: Implications of Serum and Consumable Availability for Clinicians, Researchers, and Commercial Translation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

920

764. Enhancing the Expression of Foreign Genes with Caffeine in Transfected and Infected Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 172 KB

english, 2012

921

13. Peri-Nuclear Accumulation of Adeno-Associated Virus: A Novel Barrier Limiting AAV Transduction?

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 208 KB

english, 2012

922

15. In Vivo Knock-Down of MRN Complex Members Improves Liver and Cardiac Transduction with AAV Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 208 KB

english, 2012

923

21. Nucleic Acid Carriers for In Vitro and In Vivo Delivery Based on Sequence-Defined T-Shape Polymers

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 327 KB

english, 2012

924

33. Therapeutic Applications of Sleeping Beauty System and iPS technology To Treat Duchenne Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

925

39. Improved Repair of Equine Articular Cartilage 8 Weeks Following Implantation of Chondrocytes Transduced with rAAV5-IGF-1

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 316 KB

english, 2012

926

46. Gene Therapy of Congenital Erythropoietic Porphyria Using Genetically Modified Induced Pluripotent Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

927

57. Correction of Hemophilia B Phenotype Following ZFN Mediated Genome Editing in Adult Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

928

59. AAV-Mediated Gene Repair Is Significantly Enhanced by Transient Inhibition of NHEJ or the Proteasome In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 233 KB

english, 2012

929

61. Single Nucleotide Mismatches Decrease the Frequency of Gene Targeting

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 204 KB

english, 2012

930

65. Integration of ß-Globin Expressing Lentiviral Vectors in the Human Genome Induces Alternative Splicing and the Generation of Aberrant Transcripts

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

931

66. Effective Treatment of Hemophilia A Using Hematopoietic Stem Cell Transplantation and a Bioengineered Factor VIII Transgene

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 191 KB

english, 2012

932

74. AAV Gene Therapy with Cholesterol 24-Hydroxylase for Alzheimer Disease: In Vivo Consequences on Amyloid and Tau Components of the Pathology

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 218 KB

english, 2012

933

75. AAV8-Mediated Expression of VEGF Antagonist Ranibizumab in Macaque Eye: Comparison of Subretinal vs. Intravitreal Delivery of Vector

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

934

101. CFTR Gene Correction of Only the Surface Airway Epithelium Restores Antimicrobial Activity to CFTR Knockout Ferret Tracheas Ex Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 227 KB

english, 2012

935

108. Selection and Isolation of Patient Specific AAV Nab Escape Mutants

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

936

111. AAV6 Functional Endocytosis in Mouse Embryo Fibroblast Is Independent of Clathrin and Caveolin Mechanisms and Does Not Require EGFR Activation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 176 KB

english, 2012

937

117. Pre-Existing Neutralizing Antibodies to AAV2 and AAV5 in the Serum and Synovial Fluid of 20 Horses

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 311 KB

english, 2012

938

160. Gene Therapy for Stargardt Disease Using Helper-Dependent Adenoviral Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

939

164. In Utero Brain-Directed AAV5 Gene Therapy Results in Rapid, Robust, and Specific Transduction of Choroid Plexus Epithelia: Implications for Rescue of Prenatal Lethal Mouse Models of Neurometabolic Disease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 251 KB

english, 2012

940

176. AAV-Mediated Gene Therapy in Limb-Girdle Muscular Dystrophy 2I (LGMD2I) Mouse Model

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 322 KB

english, 2012

941

177. rAAV9-Mediated Microdystrophin Gene Transfer with Immune Tolerance Induction Improves Dystrophic Phenotype of Canine X-Linked Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

942

184. Follistatin N-Terminal Mutation Diminishes Muscle Growth Effects In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 378 KB

english, 2012

943

190. Analysis of Gene Expression in Adoptively Transferred TCR-Engineered Lymphocytes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

944

198. Survey of Gene Therapy Researchers’ Perceptions and Assessment of Risks in Clinical Trials

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

945

199. HLA Typing as an Identity Test for Patients and Their FANGTM Autologous Cancer Vaccines

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

946

202. Novel Oncolytic HSV-1 for the Treatment of Prostate Carcinoma with Improved Efficacy Independent of Tumor Growth Rate

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 212 KB

english, 2012

947

204. A Phase I Dose Escalation Trial of Intratumoral Injection with Oncoloytic Adenovirus (Ad) Vector VRX-007 in Patients with Advanced Solid Tumors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

948

209. Use of Helicobacter pyroli Neutrophil Activating Protein (NAP) as an Immune-Modulatory Agent To Enhance the Efficacy of Oncolytic Adenovirus Therapy for Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 373 KB

english, 2012

949

222. Addressing the Genotoxic Potential of SIN Lentiviral Vectors for Liver Gene Transfer in Mouse Models of Hepatocarcinogenesis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

950

231. Response of Hematopoietic Cells to the Activation of Evi1

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 308 KB

english, 2012

951

245. Respiratory Tract Tropism Difference of FIV and VEV Vectors Pseudotyped with GP64 Envelope Glycoprotein

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

952

246. Functional Decline, Remodeling, and Compensation in the Respiratory System of a Canine Model of Duchenne Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

953

249. Self-Complementary AAV Vectors Delivered Intramuscularly Induce an Enhanced Immune Response to Factor IX

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

954

250. Enhanced Amplitude and Functionality of T Cell Responses in Virally Infected Human-Like Cmah Null Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 231 KB

english, 2012

955

256. A Comprehensive Study on Immunology and Persistence of AAV8 Vectors with Self-Complementary and Single-Stranded Genome in Mice: Implications for Transient Immune Suppression with AAV8 Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 222 KB

english, 2012

956

270. Protamine Containing Chitosan:shRNA Complexes for Silencing of VEGF Expression

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

957

277. Time-Dependent Effects of Synthetic Double-Stranded RNA on Eag1 Potassium Channel mRNA Content from Human Glioma Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 368 KB

english, 2012

958

281. Rapid Generation of Induced Pluripotent Stem Cells (iPSCs) from the Urine of a Patient with Duchenne Muscular Dystrophy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 501 KB

english, 2012

959

289. Generation of Footprint Free iPSCs from CD34+ and PBMCs Using Sendai Virus Vector and Application of Alkaline Phosphatase Live Stainning Dye

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

960

290. Comparison of Enzymatic and Non-Enzymatic Passaging of Human Embryonic Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

961

296. Protease-Activated Receptor 2 Cooperates with Toll-Like Receptor 4 in Sensing of Adenovirus-Coagulation Factor X Complex and Activating Innate Immune and Inflammatory Responses

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

962

305. Using Homologous Recombination To Stack Genetic Resistance to HIV

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

963

315. Hepatic Gene Transfer of TFEB Results in Clearance of Mutant alpha-1-Antitrypsin

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 175 KB

english, 2012

964

326. HCV Inhibition by a Multicistronic Anti-HCV miRNA AAV Vector In Vitro and In Vivo

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 247 KB

english, 2012

965

331. Phase I Study of the “Triad” Autologous (FANG™) Vaccine, Incorporating Bifunctional shRNAfurin and GMCSF Transgene Expression, in Advanced Cancer Patients

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 229 KB

english, 2012

966

334. AAV-Mediated CNS Gene Transfer of Bevacizumab Reduces Glioblastoma Growth and Increases Survival in Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 249 KB

english, 2012

967

343. A Novel U3 LTR Attachment Site Deleted Non-Integrating Lentiviral Vector

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 287 KB

english, 2012

968

348. A Novel Jurkat-LMO2 Assay System for Vector Safety Testing and Insulator Screening

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 370 KB

english, 2012

969

352. Efficient and Non-Toxic Targeting of a Human Safe Harbor Locus with a Lentiviral Vector Associated Meganuclease

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

970

363. Activation of the Cellular Unfolded Protein Response by Recombinant Adeno-Associated Virus Vectors: Implications in Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 299 KB

english, 2012

971

371. Systemic Gene Expression after Intravenous Injection of Adeno-Associated Virus 2/9 to Fetal and Neonatal Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 314 KB

english, 2012

972

376. An Adenovirus Serotype 5 Vector with Tat-PTD Hexon Modification and Serotype 35 Fiber Shows Greatly Enhanced Transduction Capacity of Human Primary Cell Cultures

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 418 KB

english, 2012

973

386. Large-Scale Library Production Provides New Insight into Zinc Finger Specificity

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 314 KB

english, 2012

974

387. A Novel Approach for Zinc-Finger Nuclease Assembly Using Selected Two-Finger Modules

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 522 KB

english, 2012

975

388. AAV-Mediated Targeted Gene Addition in Patient-Specific SCID-X1 Induced Pluripotent Stem Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 377 KB

english, 2012

976

390. Non-Integrating Lentiviral Vectors for Efficient and Transient Delivery of Zinc Finger Nucleases and Donor Constructs for Site-Specific Gene Correction

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 422 KB

english, 2012

977

391. Factors Affecting Maintenance of the Sleeping Beauty Transposon-Mediated Expression of Human alpha-L-Iduronidase in Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 241 KB

english, 2012

978

395. In Vivo mRNA Delivery to Central Nervous System Using Polyplex Nanomicelles

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 216 KB

english, 2012

979

404. Polyacridine Octreotide Peptides for Targeted Gene Delivery

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 246 KB

english, 2012

980

411. Chitosan-Hyaluronic Acid Hybrid Vectors for Retinal Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 350 KB

english, 2012

981

419. Effect of Exendin-4 Expressed by Two-Step Transcription Amplification Plasmid System with Arginine-Grafted Bioreducible Polymer for Type 2 Diabetes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 209 KB

english, 2012

982

443. Autologous Muscle-Derived Stem Cell Treatment for Female Stress Urinary Incontinence: A 1-Year Follow-Up of Polish Investigation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 211 KB

english, 2012

983

464. Correlation of Immune Response with Long Term Survival of Refractory Cancer Patients Who Received TGF-β2 Antisense/GM-CSF Gene Modified Autologous Tumor Cell (TAG) Vaccine

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 233 KB

english, 2012

984

467. Generation of Antigen Specific T Cells from Human Hematopoietic Stem/Progenitor Cells (HSPC) Transduced with the cDNA for a T Cell Receptor Specific for the MART-1 Antigen

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

985

472. Blockade of Bcl-2 Family Apoptosis Inhibitors Using ABT-737 (ABT-263 Analog) Potentiate CAR T Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 177 KB

english, 2012

986

473. Allele-Specific Inhibition of Nanog Family Members Inhibits Three-Dimensional (3-D) Growth of Human Colorectal Carcinoma (CRC) In Vitro

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

987

480. Angiotensin Type 2 Receptors Inhibit Growth of Hepatocellular Carcinoma through Cell Cycle Arrest and Apoptosis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

988

492. Foxp3-Retrovirus Transduction to CD4+ T Cells Induce Regulatory Potential with Decreased Secretion of Interferon-gamma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 221 KB

english, 2012

989

501. Safe and Efficient In Vivo Selection of Hematopoietic Cells Gene-Modified with Chemotherapy Inducible Methylguanine Methyltransferase P140K

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 193 KB

english, 2012

990

504. AAV Vectors for Tissue-Specific, Regulated Expression In Muscle

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 192 KB

english, 2012

991

505. Physiological Regulation of Transgene Expression by a Lentiviral Vector Containing the A2UCOE Linked to a Myeloid Promoter

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 206 KB

english, 2012

992

506. Epigenetic Status of Cell-Fate Associated Promoters during Expansion of Bone-Marrow Derived Multipotent Mesenchymal Stromal Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 183 KB

english, 2012

993

509. Deletion of Proline 45 (PDX-1 ΔP45) Enhances Oncogenic Properties of PDX-1

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 230 KB

english, 2012

994

511. Expression of GMCSF, TGFβ1, TGFβ2 and Correlation to FURIN Expression in Culture Supernatants from Pre- and Post-FANG™ Plasmid Transfected Autologous Tumor Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 230 KB

english, 2012

995

515. Ex Vivo Conditioning Regimen by Reversible Non-Muscle Myosin-II Inhibition and Aryl Hydrocarbon Receptor Antagonism Increases Platelet Recovery and Maintains Long-Term Hematopoietic Reconstitution after Human Bone Marrow Transplantation

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

996

520. Immortalized Adults Human Neural Stem Cells Differentiate into Functional Neurons after Transplantation into the Mouse Rat Brain with Intracerebral Hemorrhage

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

997

530. An AAV Vector-Based Toolbox for Somatic Reprogramming and for iPS Cell Tracking and Purging

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

998

533. Targeting Intracellular Antigens Using Chimeric Antigen Receptors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 224 KB

english, 2012

999

540. Oncolytic Properties of a Vesicular Stomatitis/Measles Virus Hybrid

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 207 KB

english, 2012

1000

554. Transcriptional Activation of PRDM16 and HMGA2 and Associated Retroviral Integration in Multiple Clones without Adverse Hematopoietic Consequences Following Transplant of Autologous MGMTP140K Gene-Modified CD34+ Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 214 KB

english, 2012

1001

555. AAV2(Y-F) Vector Substantially Reduces Targeting of Transduced Hepatocytes by Capsid-Specific CD8+ T Cells

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 196 KB

english, 2012

1002

558. Liver-Directed Gene Transfer for the Induction of Antigen-Specific Tolerance: Mechanism and Potential Therapeutic Applications

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 194 KB

english, 2012

1003

564. Development of Universal Donor Human Pluripotent Stem Cells by Engineering of the HLA Locus

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 293 KB

english, 2012

1004

573. High-Throughput Screening Identifies Effective Enhancers of Lentiviral Transduction

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 208 KB

english, 2012

1005

588. Reproducible and Scalable Production Processes of Recombinant Adeno-Associated Virus Compatible with Current Good Manufacturing Practice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 376 KB

english, 2012

1006

592. Mechanism of Transduction of Human Hematopoietic Cells by Recombinant AAV6 Vectors

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 199 KB

english, 2012

1007

594. Scalable Single-Step Affinity Purification of rAAV Vector Using AVB-Sepharose Yields High Purity and High Titer Vector

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

1008

596. Negative Regulation-Resistant p53 Variant Enhances Oncolytic Adenoviral Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

1009

627. Non-Invasive, Multimodal Imaging of Microvesicles with Metabolically Biotinylated, Membrane-Bound Gaussia Luciferase

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 357 KB

english, 2012

1010

631. Optimization of Electroporation-Enhanced Intradermal Delivery of DNA Vaccine Using a Minimally Invasive Device

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 194 KB

english, 2012

1011

632. PEG Length and Linkage Dramatically Influence PEGylated Polyacridine Peptide DNA Polyplex Pharmacokinetics and Hydrodynamically-Stimulated Gene Expression

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 194 KB

english, 2012

1012

633. Targeted Nanocomplex-Delivered ENaC siRNA as a Therapeutic Strategy for Cystic Fibrosis

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 243 KB

english, 2012

1013

637. Effect of PEG Grafting Degree on Morphology and Transfection Efficiency of Polycation-g-PEG/DNA Nanoparticles

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 377 KB

english, 2012

1014

641. Optimizing Gene Delivery Properties of PEGylated Polyacridine Peptides through Sequence Specific Structure Activity Relationships

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 381 KB

english, 2012

1015

644. Impact of Novel Biologically Active Chromophores on Common Bioassays

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 174 KB

english, 2012

1016

649. Structure-Activity Relationships in a Series of Pyridinium Gemini Surfactants for Gene Delivery Bearing Hydrophilic Linkers

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 501 KB

english, 2012

1017

650. Gold Nanoparticle Mediated Gene Delivery Via Surface Charge Reversal with Pyridinium Amphiphiles

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

1018

658. Development of a Physiologically Relevant In Vitro Model of AAV8 Transduction of Primary Mouse Hepatocytes

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 184 KB

english, 2012

1019

667. Systemic Immunization with Mucosal Chemokine Molecular Adjuvant TECK/CCL25 Targets Antigen Specific Immune Responses in the Mucosa-Associated Lymphoid Tissue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

1020

675. Prostate Cancer Immunotherapy by Direct Lentivirus Vector Injection: Murine and Rhesus Macaque Models

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 249 KB

english, 2012

1021

680. Gene Therapy of Chondroitin Sulfate-Coated Ternary GM-CSF Plasmid Complex for Ovarian Cancer

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 506 KB

english, 2012

1022

683. Exploiting Endogenous microRNA Regulation To Target Gene Therapy to Tumors by Infiltrating Macrophages

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 682 KB

english, 2012

1023

687. The First Seven Days of a Brain Tumor: Glioma Cells Spread Exclusively throughout the Perivascular Compartment of the Brain in a Galectin-1 Dependent Manner. Implications for the Treatment of Brain Tumors with Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 733 KB

english, 2012

1024

692. Multi-Optimization and Image-Aided In Vivo Evaluation of Adenovirus Containing Multi-Targeting Devices for Hepatocellular Carcinoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 197 KB

english, 2012

1025

694. Characterization of the Mechanisms of Tumor Dormancy and Recurrence Following Front Line Immuno-, Viro- or Suicide Gene-, Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 197 KB

english, 2012

1026

704. Linking Epigenetics and Gene Therapy: Enhanced Replication and Therapeutic Effect of a CRAd by Placing a Nucleosome Positioning Sequence Upstream of a Tumor Specific Promoter

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 195 KB

english, 2012

1027

713. Abstract Withdrawn

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 202 KB

english, 2012

1028

727. Efficiency of RNAi-Induced Silencing in Airway Epithelial Cells Is Reduced

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 240 KB

english, 2012

1029

743. Proliferation and Multilineage Differentiation Potential of Adipose-Derived Stem Cells Isolated from Dystrophin and Utrophin Knockout Mice

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 186 KB

english, 2012

1030

744. Recipient MSCs Induced Differential Cytokine Gene Expression in the Small Intestine Allograft of Recipient Rats

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 258 KB

english, 2012

1031

765. Cell Therapy Industry Market Characteristics: Implications for Clinicians, Investors, Patients and Translational Researchers

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 172 KB

english, 2012

1032

Corrigendum to “Hematopoietic Stem Cell Mobilization for Gene Therapy of Adult Patients With Severe β-Thalassemia: Results of Clinical Trials Using G-CSF or Plerixafor in Splenectomized and Nonsplenectomized Subjects”

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 133 KB

english, 2012

1033

1. Lentiviral Vector-Based Insertional Mutagenesis Identifies New Clinically Relevant Cancer Genes Involved in the Pathogenesis of Hepatocellular Carcinoma

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 194 KB

english, 2012

1034

Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

Journal:

Molecular Therapy

Year:

2012

File:

PDF, 39 KB

2012

1035

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 584 KB

english, 2012

1036

Human Erythropoietin Gene Delivery Using an Arginine-grafted Bioreducible Polymer System

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 175 KB

english, 2012

1037

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.08 MB

english, 2012

1038

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 552 KB

english, 2012

1039

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.09 MB

english, 2012

1040

Corrigendum: Discovery of a Linear Peptide for Improving Tumor Targeting of Gene Products and Treatment of Distal Tumors by IL-12 Gene Therapy

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 346 KB

english, 2012

1041

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 580 KB

english, 2012

1042

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.16 MB

english, 2012

1043

The ASGCT in 2012: Charting a Path to Bring Novel Biologic Therapies to the Clinic

Breakefield, Xandra O

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 740 KB

english, 2012

1044

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

Journal:

Molecular Therapy

Year:

2012

File:

PDF, 37 KB

2012

1045

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.12 MB

english, 2012

1046

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 661 KB

english, 2012

1047

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.31 MB

english, 2012

1048

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.09 MB

english, 2012

1049

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 651 KB

english, 2012

1050

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1014 KB

english, 2012

1051

Corrigendum to “Hepatitis C Virus NS3/NS4A DNA Vaccine Induces Multiepitope T Cell Responses in Rhesus Macaques Mimicking Human Immune Responses”

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 43 KB

english, 2012

1052

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.19 MB

english, 2012

1053

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 687 KB

english, 2012

1054

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 505 KB

english, 2012

1055

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 927 KB

english, 2012

1056

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 813 KB

english, 2012

1057

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 603 KB

english, 2012

1058

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 621 KB

english, 2012

1059

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 475 KB

english, 2012

1060

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 575 KB

english, 2012

1061

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 462 KB

english, 2012

1062

Research Highlights

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 529 KB

english, 2012

1063

American Society of Gene & Cell Therapy 15th Annual Meeting

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 3.11 MB

english, 2012

1064

In This Issue

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 1.03 MB

english, 2012

1065

AUTHOR INDEX

Journal:

Molecular Therapy

Year:

2012

File:

PDF, 166 KB

2012

1066

KEYWORD INDEX

Journal:

Molecular Therapy

Year:

2012

Language:

english

File:

PDF, 161 KB

english, 2012

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