A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

Micro-dystrophins are highly promising candidates for treating Duchenne muscular dystrophy, a lethal muscle disease caused by dystrophin deficiency. Here, we report robust disease rescue in the severe DBA/2J-mdx model with a neuronal nitric oxide synthase (nNOS)-binding micro-dystrophin vector. 2 ×...

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Bibliographic Details
Main Authors: Chady H. Hakim, Nalinda B. Wasala, Xiufang Pan, Kasun Kodippili, Yongping Yue, Keqing Zhang, Gang Yao, Brittney Haffner, Sean X. Duan, Julian Ramos, Joel S. Schneider, N. Nora Yang, Jeffrey S. Chamberlain, Dongsheng Duan
Format: Article
Language:English
Published: Elsevier 2017-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
DMD
micro-dystrophin
mdx
DBA/2J
nNOS
cardiomyopathy
systemic gene therapy
Duchenne muscular dystrophy
adeno-associated virus
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050117300815

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http://www.sciencedirect.com/science/article/pii/S2329050117300815

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