Duchenne muscular dystrophy: CRISPR/Cas9 treatment

Cell Res. 2016 May;26(5):513-4. doi: 10.1038/cr.2016.28. Epub 2016 Mar 1.

Abstract

A novel approach to gene correction by genome editing shows great promise as a treatment for Duchenne muscular dystrophy (DMD). CRISPR/Cas9 delivered by adeno-associated virus to a mouse model for DMD demonstrated improvement in function and histology.

Publication types

  • Comment

MeSH terms

  • Animals
  • CRISPR-Cas Systems
  • Dystrophin / genetics
  • Genetic Therapy
  • Humans
  • Infant, Newborn
  • Mice
  • Muscular Dystrophy, Duchenne / genetics*
  • Neonatal Screening

Substances

  • Dystrophin