Volume 26, No 5, May 2016

ISSN: 1001-0602 
EISSN: 1748-7838 2018 
impact factor 17.848* 
(Clarivate Analytics, 2019)

Volume 26 Issue 5, May 2016: 513-514

RESEARCH HIGHLIGHTS

Duchenne muscular dystrophy: CRISPR/Cas9 treatment

Jerry R Mendell^1,2,3 and Louise R Rodino-Klapac^1,2,3

¹Department of Pediatrics, Columbus, OH 43205, USA
²Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA
³The Ohio State University, Columbus, OH 43205, USA Correspondence: (Jerry.Mendell@nationwidechildrens.org)

A novel approach to gene correction by genome editing shows great promise as a treatment for Duchenne muscular dystrophy (DMD). CRISPR/Cas9 delivered by adeno-associated virus to a mouse model for DMD demonstrated improvement in function and histology.

10.1038/cr.2016.28

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