Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

CRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrophin expression in skeletal and cardiac muscle in m...

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Autores principales: Niclas E. Bengtsson, John K. Hall, Guy L. Odom, Michael P. Phelps, Colin R. Andrus, R. David Hawkins, Stephen D. Hauschka, Joel R. Chamberlain, Jeffrey S. Chamberlain
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2017
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Science
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Acceso en línea:https://doaj.org/article/42b84f4d2d7e485cb3c0f8f3e7eaf353
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