Open AccessCRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells
Author(s) -
Yi-Li Min,
Hui Li,
Cristina Rodríguez-Caycedo,
Alex A. Mireault,
Jian Huang,
John M. Shelton,
John McAnally,
Leonela Amoasii,
Pradeep P.A. Mammen,
Rhonda BasselDuby,
Eric N. Olson
Publication year - 2019
Publication title -
science advances
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 5.928
H-Index - 146
ISSN - 2375-2548
DOI - 10.1126/sciadv.aav4324
Subject(s) - crispr , duchenne muscular dystrophy , exon , genome editing , cas9 , gene , genetics , biology , mutation , muscular dystrophy , dystrophin
A mouse model with a common human DMD mutation was created and corrected by CRISPR-Cas9 gene editing.
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