CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice

Duchenne muscular dystrophy (DMD), caused by mutations in the X-linked dystrophin gene (DMD), is characterized by fatal degeneration of striated muscles. Dilated cardiomyopathy is one of the most common lethal features of the disease. We deployed Cpf1, a unique class 2 CRISPR (clustered regularly in...

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Veröffentlicht in:Sci Adv
Hauptverfasser: Zhang, Yu, Long, Chengzu, Li, Hui, McAnally, John R., Baskin, Kedryn K., Shelton, John M., Bassel-Duby, Rhonda, Olson, Eric N.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Association for the Advancement of Science 2017
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Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5389745/
https://ncbi.nlm.nih.gov/pubmed/28439558
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.1602814
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