The promise and challenge of therapeutic genome editing

Nature. 2020 Feb;578(7794):229-236. doi: 10.1038/s41586-020-1978-5. Epub 2020 Feb 12.


Genome editing, which involves the precise manipulation of cellular DNA sequences to alter cell fates and organism traits, has the potential to both improve our understanding of human genetics and cure genetic disease. Here I discuss the scientific, technical and ethical aspects of using CRISPR (clustered regularly interspaced short palindromic repeats) technology for therapeutic applications in humans, focusing on specific examples that highlight both opportunities and challenges. Genome editing is-or will soon be-in the clinic for several diseases, with more applications under development. The rapid pace of the field demands active efforts to ensure that this breakthrough technology is used responsibly to treat, cure and prevent genetic disease.

Publication types

  • Review

MeSH terms

  • Anemia, Sickle Cell / genetics*
  • Anemia, Sickle Cell / therapy*
  • CRISPR-Cas Systems / genetics
  • Gene Editing / ethics
  • Gene Editing / methods*
  • Gene Editing / standards
  • Gene Editing / trends*
  • Genome, Human / genetics*
  • Germ-Line Mutation / genetics
  • Humans
  • Muscular Dystrophy, Duchenne / genetics*
  • Muscular Dystrophy, Duchenne / therapy*
  • Organ Specificity / genetics
  • Patient Safety
  • beta-Globins / genetics


  • beta-Globins