Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

Autor: Niclas E. Bengtsson, John K. Hall, Guy L. Odom, Michael P. Phelps, Colin R. Andrus, R. David Hawkins, Stephen D. Hauschka, Joel R. Chamberlain, Jeffrey S. Chamberlain
Jazyk: angličtina
Rok vydání: 2017
Předmět:
Zdroj: Nature Communications, Vol 8, Iss 1, Pp 1-10 (2017)
Druh dokumentu: article
ISSN: 2041-1723
DOI: 10.1038/ncomms14454
Popis: CRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrophin expression in skeletal and cardiac muscle in mice.
Databáze: Directory of Open Access Journals