Clinical progress in genome-editing technology and in vivo delivery techniques

Trends Genet. 2023 Mar;39(3):208-216. doi: 10.1016/j.tig.2022.12.001. Epub 2023 Jan 18.


There is wide interest in applying genome-editing tools to prevent, treat, and cure a variety of diseases. Since the discovery of the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas) systems, these techniques have been used in combination with different delivery systems to create highly efficacious treatment options. Each delivery system has its own advantages and disadvantages and is being used for various applications. With the large number of gene-editing applications being studied but very few being brought into the clinic, we review current progress in the field, specifically where genome editing has been applied in vivo and in the clinic, and identify current challenges and areas of future growth.

Keywords: CRISPR/Cas9; clinical trials; delivery systems; genome editing; in vivo.

Publication types

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

MeSH terms

  • CRISPR-Cas Systems*
  • Gene Editing* / methods
  • Treatment Outcome