CRISPR-Cas9 base editors and their current role in human therapeutics

Cytotherapy. 2023 Mar;25(3):270-276. doi: 10.1016/j.jcyt.2022.11.013. Epub 2023 Jan 10.


Background: Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification.

Methods: This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes.

Results: CRISPR-Cas9 BEs are a useful system because of their highefficiency and broad applicability to gene correction and disruption. In addition, base editing has beensuggested as a safer approach than other CRISPR-Cas9-based systems, as it limits double-strand breaksduring multiplex gene knockout and does not require a toxic DNA donor molecule for genetic correction.

Conclusion: As such, numerous industry and academic groups are currently developing base editing strategies withclinical applications in cancer immunotherapy and gene therapy, which this review will discuss, with a focuson current and future applications of in vivo BE delivery.

Keywords: base editor; cancer immunotherapy; gene therapy; hematopoietic stem cell; multiplex gene editing; sickle cell.

Publication types

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

MeSH terms

  • CRISPR-Cas Systems* / genetics
  • DNA
  • Gene Editing*
  • Genetic Therapy
  • Humans


  • BES
  • DNA