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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Gepubliceerd in:Mol Ther Methods Clin Dev
Hoofdauteurs: Hakim, Chady H., Wasala, Nalinda B., Pan, Xiufang, Kodippili, Kasun, Yue, Yongping, Zhang, Keqing, Yao, Gang, Haffner, Brittney, Duan, Sean X., Ramos, Julian, Schneider, Joel S., Yang, N. Nora, Chamberlain, Jeffrey S., Duan, Dongsheng
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Gene & Cell Therapy 2017
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Original Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5596503/
https://ncbi.nlm.nih.gov/pubmed/28932757
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.06.006
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