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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Dades bibliogràfiques
Publicat a:Mol Ther Methods Clin Dev
Autors principals: 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
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2017
Matèries:
Original Article
Accés en línia: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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