Functional correction of dystrophin actin binding domain mutations by genome editing

Dystrophin maintains the integrity of striated muscles by linking the actin cytoskeleton with the cell membrane. Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene (DMD) that result in progressive, debilitating muscle weakness, cardiomyopathy, and a shortened lifespan. M...

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Detalhes bibliográficos
Publicado no:JCI Insight
Main Authors: Kyrychenko, Viktoriia, Kyrychenko, Sergii, Tiburcy, Malte, Shelton, John M., Long, Chengzu, Schneider, Jay W., Zimmermann, Wolfram-Hubertus, Bassel-Duby, Rhonda, Olson, Eric N.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2017
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5621913/
https://ncbi.nlm.nih.gov/pubmed/28931764
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.95918
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