Journal
CELL RESEARCH
Volume 26, Issue 5, Pages 513-514Publisher
INST BIOCHEMISTRY & CELL BIOLOGY
DOI: 10.1038/cr.2016.28
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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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