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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...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
The Company of Biologists Limited
2015
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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 |