title: Exon Snipping in Duchenne Muscular Dystrophy.
abstract: 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.
authors: Genetics and Genome Biology Program, the Hospital for Sick Children, Toronto, ON, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON, Canada.
authors: Dwi U
authors: DU
authors: Kemaladewi
authors: 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: ronald.cohn@sickkids.ca.
authors: Ronald D
authors: RD
authors: Cohn
mesh_terms: D000818:Animals; D064113:CRISPR-Cas Systems; D016189:Dystrophin / Q000235:genetics*; D005091:Exons / Q000235:genetics*; D015316:Genetic Therapy / Q000379:methods*; D008297:Male; D018482:Muscle, Skeletal / Q000378:metabolism*; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy*
chemical_list: D016189:Dystrophin