Illuminating the genome-wide activity of genome editors for safe and effective therapeutics

Genome Biol. 2018 Dec 22;19(1):226. doi: 10.1186/s13059-018-1610-2.


Genome editing holds remarkable promise to transform human medicine as new therapies that can directly address the genetic causes of disease. However, concerns remain about possible undesired biological consequences of genome editors, particularly the introduction of unintended 'off-target' mutations. Here, we discuss both important considerations for therapeutic genome editing and our understanding of the functional impact of undesired off-target mutations. An important challenge for the future will be the development of new approaches for predicting and defining the probable function of unintended genome-editing mutations, which will inspire confidence in the next generation of promising genome-editing therapies.

Publication types

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

MeSH terms

  • Gene Editing*
  • Genetic Therapy*
  • Humans
  • Mutation
  • Patient Safety