Volume 22; Issue 4

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Molecular Therapy
Volume 22; Issue 4

Molecular Therapy

Volume 22; Issue 4

1

Dual Masking of Specific Negative Splicing Regulatory Elements Resulted in Maximal Exon 7 Inclusion of SMN2 Gene

Pao, Peng Wen, Wee, Keng Boon, Yee, Woon Chee, DwiPramono, Zacharias Aloysius

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 699 KB

english, 2014

2

Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant

Melo, Sandra P, Lisowski, Leszek, Bashkirova, Elizaveta, Zhen, Hanson H, Chu, Kirk, Keene, Douglas R, Marinkovich, M Peter, Kay, Mark A, Oro, Anthony E

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 1.21 MB

english, 2014

3

Repairing Without Cutting: A Safer Alternative to Gene Correction?

Mavilio, Fulvio

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 145 KB

english, 2014

4

Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice

ElMallah, Mai K, Falk, Darin J, Nayak, Sushrusha, Federico, Roland A, Sandhu, Milapjit S, Poirier, Amy, Byrne, Barry J, Fuller, David D

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 1.77 MB

english, 2014

5

Corrective GUSB Transfer to the Canine Mucopolysaccharidosis VII Brain

Cubizolle, Aurelie, Serratrice, Nicolas, Skander, Nadia, Colle, Marie-Anne, Ibanes, Sandy, Gennetier, Aurelie, Bayo-Puxan, Neus, Mazouni, Khalil, Mennechet, Franck, Joussemet, Beatrice, Cherel, Yan, L

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 2.05 MB

english, 2014

6

Targeting Estrogen-Related Receptor Alpha Inhibits Epithelial-to-Mesenchymal Transition and Stem Cell Properties of Ovarian Cancer Cells

Lam, Sophia SN, Mak, Abby SC, Yam, Judy WP, Cheung, Annie NY, Ngan, Hextan YS, Wong, Alice ST

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 734 KB

english, 2014

7

Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo

Cesana, Daniela, Ranzani, Marco, Volpin, Monica, Bartholomae, Cynthia, Duros, Caroline, Artus, Alexandre, Merella, Stefania, Benedicenti, Fabrizio, Sergi Sergi, Lucia, Sanvito, Francesca, Brombin, Chi

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 1.30 MB

english, 2014

8

MicroRNA-29b Inhibits Diabetic Nephropathy in db/db Mice

Chen, Hai-Yong, Zhong, Xiang, Huang, Xiao R, Meng, Xiao-Ming, You, Yongke, Chung, Arthur CK, Lan, Hui Y

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 1.87 MB

english, 2014

9

Skin Electroporation of a Plasmid Encoding hCAP-18/LL-37 Host Defense Peptide Promotes Wound Healing

Steinstraesser, Lars, Lam, Martin C, Jacobsen, Frank, Porporato, Paolo E, Chereddy, Kiran Kumar, Becerikli, Mustafa, Stricker, Ingo, Hancock, Robert EW, Lehnhardt, Marcus, Sonveaux, Pierre, Préat, Vér

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 1.30 MB

english, 2014

10

Conditioning the Cochlea to Facilitate Survival and Integration of Exogenous Cells into the Auditory Epithelium

Park, Yong-Ho, Wilson, Kevin F., Ueda, Yoshihisa, Tung Wong, Hiu, Beyer, Lisa A., Swiderski, Donald L., Dolan, David F., Raphael, Yehoash

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 1.51 MB

english, 2014

11

Delivery of Therapeutic siRNA to the Lung Endothelium via Novel Lipoplex Formulation DACC

Fehring, V, Schaeper, U, Ahrens, K, Santel, A, Keil, O, Eisermann, M, Giese, K, Kaufmann, Jörg

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 912 KB

english, 2014

12

Alphavirus-based Vaccines Encoding Nonstructural Proteins of Hepatitis C Virus Induce Robust and Protective T-cell Responses

Ip, Peng Peng, Boerma, Annemarie, Regts, Joke, Meijerhof, Tjarko, Wilschut, Jan, Nijman, Hans W, Daemen, Toos

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 634 KB

english, 2014

13

Recombinant AAV as a Platform for Translating the Therapeutic Potential of RNA Interference

Borel, Florie, Kay, Mark A, Mueller, Christian

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 709 KB

english, 2014

14

Vascular Delivery of rAAVrh74.MCK.GALGT2 to the Gastrocnemius Muscle of the Rhesus Macaque Stimulates the Expression of Dystrophin and Laminin α2 Surrogates

Chicoine, Louis G., Rodino-Klapac, Louise R., Shao, Guohong, Xu, Rui, Bremer, William G., Camboni, Marybeth, Golden, Bethannie, Montgomery, Chrystal L., Shontz, Kimberly, Heller, Kristin N., Griffin,

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 1.31 MB

english, 2014

15

Research Highlights

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 111 KB

english, 2014

16

Targeting the Splicing of mRNA in Autoimmune Diseases: BAFF Inhibition in Sjögren’s Syndrome as a Proof of Concept

Roescher, N., Vosters, J.L., Alsaleh, G., Dreyfus, P., Jacques, S., Chiocchia, G., Sibilia, J., Tak, P.P., Chiorini, J.A., Mariette, X., Gottenberg, Jacques-Eric

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 676 KB

english, 2014

17

Intrajugular Vein Delivery of AAV9-RNAi Prevents Neuropathological Changes and Weight Loss in Huntington’s Disease Mice

Dufour, Brett D, Smith, Catherine A, Clark, Randall L, Walker, Timothy R, McBride, Jodi L

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 2.23 MB

english, 2014

18

Strengthening the Skin with Topical Delivery of Keratinocyte Growth Factor-1 Using a Novel DNA Plasmid

Dou, Chunqing, Lay, Frank, Ansari, Amir Mehdi, Rees, Donald J, Ahmed, Ali Karim, Kovbasnjuk, Olga, Matsangos, Aerielle E., Du, Junkai, Hosseini, Sayed Mohammad, Steenbergen, Charles, Fox-Talbot, Karen

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 1.39 MB

english, 2014

19

The Evolution of Gene Transfer, Gene Therapy, and the RAC: IOM Recommendations to the NIH Director

Federoff, Howard J, Wagner, John E

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 135 KB

english, 2014

20

Corrigendum to “Dual Masking of Specific Negative Splicing Regulatory Elements Resulted in Maximal Exon 7 Inclusion of SMN2 Gene”

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 44 KB

english, 2014

21

Corrigendum to “Silencing Mutant ATXN3 Expression Resolves Molecular Phenotypes in SCA3 Transgenic Mice”

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 44 KB

english, 2014

22

In This Issue

Journal:

Molecular Therapy

Year:

2014

Language:

english

File:

PDF, 975 KB

english, 2014

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