CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

Physiology (Bethesda). 2019 Sep 1;34(5):341-353. doi: 10.1152/physiol.00012.2019.


This is a review describing advances in CRISPR/Cas-mediated therapies for neuromuscular disorders (NMDs). We explore both CRISPR-mediated editing and dead Cas approaches as potential therapeutic strategies for multiple NMDs. Last, therapeutic considerations, including delivery and off-target effects, are also discussed.

Keywords: CRISPR/Cas; gene editing; gene therapy; muscular dystrophy; neuromuscular disorders.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics*
  • Gene Editing / methods
  • Humans
  • Neuromuscular Diseases / genetics*