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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Detalles Bibliográficos
Autores principales: Zhang, Yu, Long, Chengzu, Li, Hui, McAnally, John R., Baskin, Kedryn K., Shelton, John M., Bassel-Duby, Rhonda, Olson, Eric N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2017
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5389745/
https://www.ncbi.nlm.nih.gov/pubmed/28439558
http://dx.doi.org/10.1126/sciadv.1602814

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5389745/
https://www.ncbi.nlm.nih.gov/pubmed/28439558
http://dx.doi.org/10.1126/sciadv.1602814

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