Duchenne muscular dystrophy: CRISPR/Cas9 treatment

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Abstract

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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Mendell, J. R., & Rodino-Klapac, L. R. (2016, May 1). Duchenne muscular dystrophy: CRISPR/Cas9 treatment. Cell Research. Nature Publishing Group. https://doi.org/10.1038/cr.2016.28

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