Imprecision Medicine: A One-Size-Fits-Many Approach for Muscle Dystrophy

Cell Stem Cell. 2016 Apr 7;18(4):423-4. doi: 10.1016/j.stem.2016.03.004.


There is still no curative treatment for Duchenne muscular dystrophy (DMD). In this issue of Cell Stem Cell, Young et al. (2016) demonstrate a genome editing approach applicable to 60% of DMD patients with CRISPR/Cas9 using one pair of guide RNAs.

Publication types

  • Comment

MeSH terms

  • CRISPR-Cas Systems*
  • Dystrophin / metabolism*
  • Exons
  • Genetic Therapy
  • Humans
  • Muscular Dystrophy, Duchenne


  • Dystrophin