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Cell Res. 2016 May;26(5):513-4. doi: 10.1038/cr.2016.28. Epub 2016 Mar 1.

Duchenne muscular dystrophy: CRISPR/Cas9 treatment.

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Department of Pediatrics, Columbus, OH 43205, USA.
Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
The Ohio State University, Columbus, OH 43205, USA.


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.

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