Exon Snipping in Duchenne Muscular Dystrophy

Dwi U. Kemaladewi, Ronald D. Cohn

Research output: Contribution to journalShort surveypeer-review

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.

Original languageEnglish (US)
Pages (from-to)187-189
Number of pages3
JournalTrends in Molecular Medicine
Volume22
Issue number3
DOIs
StatePublished - Mar 1 2016

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology

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