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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Enregistré dans:
Détails bibliographiques
Publié dans:Mol Ther Methods Clin Dev
Auteurs principaux: 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
Format: Artigo
Langue:Inglês
Publié: American Society of Gene & Cell Therapy 2017
Sujets:
Original Article
Accès en ligne: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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