Exon Snipping in Duchenne Muscular Dystrophy

Trends Mol Med. 2016 Mar;22(3):187-189. doi: 10.1016/j.molmed.2016.01.007. Epub 2016 Feb 5.


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.

Publication types

  • Comment

MeSH terms

  • Animals
  • CRISPR-Cas Systems*
  • Dystrophin / genetics*
  • Exons / genetics*
  • Genetic Therapy / methods*
  • Male
  • Muscle, Skeletal / metabolism*
  • Muscular Dystrophy, Duchenne / therapy*


  • Dystrophin