Making Gene Editing a Therapeutic Reality

F1000Res. 2018 Dec 21;7:F1000 Faculty Rev-1970. doi: 10.12688/f1000research.16106.1. eCollection 2018.


This review discusses current bottlenecks in making CRISPR-Cas9-mediated genome editing a therapeutic reality and it outlines recent strategies that aim to overcome these hurdles as well as the scope of current clinical trials that pioneer the medical translation of CRISPR-Cas9. Additionally, this review outlines the specifics of disease-modifying gene editing in recessive versus dominant genetic diseases with the focus on genetic myopathies that are exemplified by Duchenne muscular dystrophy and myotonic dystrophies.

Keywords: AAV; Alpha1 Antitrypsin deficiency; CRISPR; Clinical trials; DMD; DNA damage; HDR; Myotonic Dystrophy; NHEJ; nanoparticles.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems / genetics
  • Gene Editing / methods*
  • Genetic Therapy / methods*
  • Humans
  • Muscular Dystrophy, Duchenne / genetics
  • Myotonic Dystrophy / genetics