Exon Snipping in Duchenne Muscular Dystrophy. Issue 3 (March 2016)

• Record Type: Journal Article
• Authors: Kemaladewi, Dwi U.; Cohn, Ronald D.
• Is Part Of: Trends in molecular medicine. Volume 22:Issue 3(2016)
• Page Start: 187
• Page End: 189
• Publication Date: 2016-03
• Subjects: Molecular biology -- Periodicals; Pathology, Molecular -- Periodicals; Physiology, Pathological -- Periodicals; 572.8
• DOI: 10.1016/j.molmed.2016.01.007
• Languages: English
• ISSNs: 1471-4914

Abstract

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.