CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice
Yu Zhang(Southwestern Medical Center), Chengzu Long(Southwestern Medical Center), Hui Li(Southwestern Medical Center), John McAnally(Southwestern Medical Center), Kedryn K. Baskin(Southwestern Medical Center), John M. Shelton(The University of Texas Southwestern Medical Center), Rhonda Bassel‐Duby(Southwestern Medical Center), Eric N. Olson(Southwestern Medical Center)
Cited by 240Open Access
Abstract
mice following Cpf1-mediated germline editing. These findings are the first to show the efficiency of Cpf1-mediated correction of genetic mutations in human cells and an animal disease model and represent a significant step toward therapeutic translation of gene editing for correction of DMD.
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