Title: | CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice |
Journal : | Science advances, 3, 4 |
Authors: | Zhang Y, Author ; Long C ; Li H ; McAnally JR ; Baskin KK ; Shelton JM ; Bassel Duby R ; Olson EN |
Material Type: | Article |
Publication Date: | 2017 |
Languages: | English |
Keywords : | CRISPR/Cpf1 ; Duchenne muscular dystrophy ; DYS gene ; dystrophin ; exon skipping ; genetic mutations ; genome editing ; heart muscle fibers ; induced pluripotent stem cells ; mdx mouse ; reading frame restoration |
Keywords: | Duchenne muscular dystrophy;Skeletal muscle;dystrophin;exon skipping;guide RNA;iPSC; |
Pubmed / DOI : | DOI : 10.1126/sciadv.1602814 / Pubmed : 28439558 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/28439558 |