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

Dawn E Bowles; Scott WJ McPhee; Chengwen Li; Steven J Gray; Jade J Samulski; Angelique S Camp; Juan Li; Bing Wang; Paul E Monahan; Joseph E Rabinowitz; Joshua C Grieger; Lakshmanan Govindasamy; Mavis Agbandje-McKenna; Xiao Xiao; R Jude Samulski

doi:10.1038/mt.2011.237

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

Molecular Therapy ◽

10.1038/mt.2011.237 ◽

2012 ◽

Vol 20 (2) ◽

pp. 443-455 ◽

Cited By ~ 223

Author(s):

Dawn E Bowles ◽

Scott WJ McPhee ◽

Chengwen Li ◽

Steven J Gray ◽

Jade J Samulski ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Phase 1 ◽

Aav Vector

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Efficient AAV Vector Production System: Towards Gene Therapy For Duchenne Muscular Dystrophy

Gene Therapy - Tools and Potential Applications ◽

10.5772/53023 ◽

2013 ◽

Cited By ~ 1

Author(s):

Takashi Okada

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Production System ◽

Aav Vector

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Advanced Gene Therapy for Treatment of Cardiomyopathy and Respiratory Insufficiency in Duchenne Muscular Dystrophy

10.21236/ada613171 ◽

2014 ◽

Author(s):

Barry J. Byrne

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Respiratory Insufficiency

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Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy

Molecular Therapy — Methods & Clinical Development ◽

10.1016/j.omtm.2020.06.016 ◽

2020 ◽

Vol 18 ◽

pp. 415-421 ◽

Cited By ~ 3

Author(s):

Virginie Mariot ◽

Caroline Le Guiner ◽

Inès Barthélémy ◽

Marie Montus ◽

Stéphane Blot ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy

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92. Safety and Histological Signs of Efficacy Following Systemic AAV9-CMV-Microutrophin Gene Therapy in a Canine Model for Duchenne Muscular Dystrophy

Molecular Therapy ◽

10.1016/s1525-0016(16)34427-6 ◽

2013 ◽

Vol 21 ◽

pp. S38

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Canine Model

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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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Dystrophin mRNA Processing in the Canine Homologue of Duchenne Muscular Dystrophy: An Authentic Model for Gene Therapy

Research and Perspectives in Neurosciences - Gene Transfer and Therapy in the Nervous System ◽

10.1007/978-3-642-84842-1_12 ◽

1992 ◽

pp. 146-157

Author(s):

N. J. H. Sharp ◽

J. N. Kornegay ◽

S. D. van Camp ◽

M. H. Herbstreith ◽

S. L. Secore ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Mrna Processing

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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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AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in >21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy

Journal of Molecular and Cellular Cardiology ◽

10.1016/j.yjmcc.2012.05.002 ◽

2012 ◽

Vol 53 (2) ◽

pp. 217-222 ◽

Cited By ~ 44

Author(s):

Brian Bostick ◽

Jin-Hong Shin ◽

Yongping Yue ◽

Nalinda B. Wasala ◽

Yi Lai ◽

...

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Myocardial Fibrosis ◽

Cardiac Death ◽

Dystrophin Gene ◽

Mdx Mice ◽

Stage Model ◽

End Stage ◽

Duchenne Muscular Dystrophy Cardiomyopathy

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1131. A Variant of Human Dihydrofolate Reductase Can Be Used To Select Myoblasts for Ex Vivo Gene Therapy of Duchenne Muscular Dystrophy

Molecular Therapy ◽

10.1016/s1525-0016(16)43961-4 ◽

2002 ◽

Vol 5 (5) ◽

pp. S367-S368

Keyword(s):

Gene Therapy ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Dihydrofolate Reductase ◽

Ex Vivo ◽

Ex Vivo Gene Therapy ◽

Human Dihydrofolate Reductase

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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

Download Full-text