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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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| Vydáno v: | Sci Adv |
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| Hlavní autoři: | , , , , , , , |
| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
American Association for the Advancement of Science
2017
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| Témata: | |
| On-line přístup: | 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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