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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Опубликовано в: :JCI Insight
Главные авторы: Kyrychenko, Viktoriia, Kyrychenko, Sergii, Tiburcy, Malte, Shelton, John M., Long, Chengzu, Schneider, Jay W., Zimmermann, Wolfram-Hubertus, Bassel-Duby, Rhonda, Olson, Eric N.
Формат: Artigo
Язык:Inglês
Опубликовано: American Society for Clinical Investigation 2017
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Research Article
Online-ссылка: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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