Exon Snipping in Duchenne Muscular Dystrophy.

Trends Mol Med

Genetics and Genome Biology Program, the Hospital for Sick Children, Toronto, ON, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON, Canada; Department of Pediatrics, University of Toronto, Toronto, ON, Canada; Division of Clinical and Metabolic Genetics, the Hospital for Sick Children, Toronto, ON, Canada; Centre for Genetic Medicine, the Hospital for Sick Children, Toronto, ON, Canada. Electronic address:

Published: March 2016

Duchenne muscular dystrophy (DMD) is a life-limiting neuromuscular disorder caused by mutations in the DMD gene encoding dystrophin. We discuss very recent studies that used CRISPR/Cas9 technology to 'snip out' mutated exons in DMD, restoring the reading frame of the gene. We also present cautionary aspects of translating this exciting technology into clinical practice.

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http://dx.doi.org/10.1016/j.molmed.2016.01.007	DOI Listing

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