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Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly promising therapeutic strategy is to replace or repair the defective dystrophin gene by gene therapy. Numerous animal m...

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Detalles Bibliográficos
Autores principales: McGreevy, Joe W., Hakim, Chady H., McIntosh, Mark A., Duan, Dongsheng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Company of Biologists Limited 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4348559/
https://www.ncbi.nlm.nih.gov/pubmed/25740330
http://dx.doi.org/10.1242/dmm.018424