Therapeutic in vivo delivery of gene editing agents

Cell. 2022 Jul 21;185(15):2806-2827. doi: 10.1016/j.cell.2022.03.045. Epub 2022 Jul 6.


In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each method and highlight opportunities for future improvements.

Publication types

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

MeSH terms

  • CRISPR-Cas Systems / genetics
  • Gene Editing* / methods
  • Genetic Therapy / methods
  • Genetic Vectors
  • Liposomes
  • Nanoparticles*


  • Lipid Nanoparticles
  • Liposomes