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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5596503/
https://www.ncbi.nlm.nih.gov/pubmed/28932757
http://dx.doi.org/10.1016/j.omtm.2017.06.006

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5596503/
https://www.ncbi.nlm.nih.gov/pubmed/28932757
http://dx.doi.org/10.1016/j.omtm.2017.06.006

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