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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Bibliografski detalji
Izdano u:Mol Ther Methods Clin Dev
Glavni autori: 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
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2017
Teme:
Original Article
Online pristup: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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