Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy

Michael E. Nance; Dongsheng Duan

doi:10.1089/hum.2015.107

Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy

Human Gene Therapy ◽

10.1089/hum.2015.107 ◽

2015 ◽

Vol 26 (12) ◽

pp. 786-800 ◽

Cited By ~ 23

Author(s):

Michael E. Nance ◽

Dongsheng Duan

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Virus Capsid ◽

Adeno Associated Virus ◽

Duchenne Muscular Dystrophy Gene

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Codon Optimization of the Microdystrophin Gene for Duchenne Muscular Dystrophy Gene Therapy

Methods in Molecular Biology - Muscle Gene Therapy ◽

10.1007/978-1-61737-982-6_2 ◽

2010 ◽

pp. 21-37 ◽

Cited By ~ 12

Author(s):

Takis Athanasopoulos ◽

Helen Foster ◽

Keith Foster ◽

George Dickson

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Codon Optimization ◽

Duchenne Muscular Dystrophy Gene

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790. Evaluation of Immune Responses to Dystrophin in Humanized mdx HLA-A*0201 Dystrophic Mice: Application in Duchenne Muscular Dystrophy Gene Therapy after U7 snRNA-Mediated Exon Skipping

Molecular Therapy ◽

10.1016/j.ymthe.2005.07.328 ◽

2005 ◽

Vol 11 ◽

pp. S307 ◽

Cited By ~ 1

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Immune Responses ◽

Exon Skipping ◽

Duchenne Muscular Dystrophy Gene ◽

Dystrophic Mice

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Engineering Exon-Skipping Vectors Expressing U7 snRNA Constructs for Duchenne Muscular Dystrophy Gene Therapy

Methods in Molecular Biology - Muscle Gene Therapy ◽

10.1007/978-1-61737-982-6_11 ◽

2010 ◽

pp. 179-196 ◽

Cited By ~ 3

Author(s):

Aurélie Goyenvalle ◽

Kay E. Davies

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Exon Skipping ◽

Duchenne Muscular Dystrophy Gene

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Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)

Neuromuscular Disorders ◽

10.1016/j.nmd.2015.06.458 ◽

2015 ◽

Vol 25 ◽

pp. S315 ◽

Cited By ~ 3

Author(s):

C. Le Guiner ◽

L. Servais ◽

M. Montus ◽

F. Bodvael ◽

B. Gjata ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Muscle Function ◽

Canine Model ◽

Virus Vector ◽

Adeno Associated Virus

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The IVth workshop on Duchenne muscular dystrophy gene therapy

The Journal of Gene Medicine ◽

10.1002/(sici)1521-2254(199911/12)1:6<444::aid-jgm74>3.0.co;2-8 ◽

1999 ◽

Vol 1 (6) ◽

pp. 444-448

Author(s):

K. Schwartz ◽

F. Leterrier

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Duchenne Muscular Dystrophy Gene

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503. Adeno-Associated Virus Vector (AAV) Microdystrophin Gene Therapy Prolongs Survival and Restores Muscle Function in the Canine Model of Duchenne Muscular Dystrophy (DMD)

Molecular Therapy ◽

10.1016/s1525-0016(16)33312-3 ◽

2016 ◽

Vol 24 ◽

pp. S200 ◽

Cited By ~ 1

Author(s):

Caroline Le Guiner ◽

Laurent Servais ◽

Marie Montus ◽

Thibaut Larcher ◽

Bodvael Fraysse ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Muscle Function ◽

Canine Model ◽

Virus Vector ◽

Adeno Associated Virus

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Duchenne muscular dystrophy gene therapy: Lost in translation?

Research and Reports in Biology ◽

10.2147/rrb.s13463 ◽

2011 ◽

pp. 31 ◽

Cited By ~ 32

Author(s):

Dongsheng Duan

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Duchenne Muscular Dystrophy Gene ◽

Lost In Translation

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Advances in Duchenne muscular dystrophy gene therapy

Nature Reviews Genetics ◽

10.1038/nrg1180 ◽

2003 ◽

Vol 4 (10) ◽

pp. 774-783 ◽

Cited By ~ 142

Author(s):

Judith C. T. van Deutekom ◽

Gert-Jan B. van Ommen

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Duchenne Muscular Dystrophy Gene

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Duchenne Muscular Dystrophy Gene Therapy in the Canine Model

Human Gene Therapy Clinical Development ◽

10.1089/humc.2015.006 ◽

2015 ◽

Vol 26 (1) ◽

pp. 57-69 ◽

Cited By ~ 37

Author(s):

Dongsheng Duan

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Canine Model ◽

Duchenne Muscular Dystrophy Gene

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688. Novel Dual AAV Vectors To Express an nNOS-Recruiting Mini-Dystrophin Gene for Duchenne Muscular Dystrophy Gene Therapy

Molecular Therapy ◽

10.1016/s1525-0016(16)37261-6 ◽

2011 ◽

Vol 19 ◽

pp. S263-S264

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Dystrophin Gene ◽

Duchenne Muscular Dystrophy Gene

Download Full-text