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

Walker S Lahr, Christopher J Sipe, Joseph G Skeate, Beau R. Webber, Branden S. Moriarity

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish (US)
Pages (from-to)270-276
Number of pages7
Issue number3
StatePublished - Mar 2023

Bibliographical note

Funding Information:
CJS was funded by the University of Minnesota's Stem Cell Institute INFUSE pre-doctoral award. JGS is supported by the T32HL007062-46 Hematology Research Training Program. BRW acknowledges funding from the National Institutes of Health (grant nos. R21CA237789, R21AI163731 and P01CA254849), Alex's Lemonade Stand Foundation, Children's Cancer Research Fund and Rein in Sarcoma. BSM acknowledges funding from the National Institutes of Health (grant nos. R01AI161017, R01AI161017, P01CA254849 and P50CA136393), Children's Cancer Research Fund and Fanconi Anemia Research Fund.

Publisher Copyright:
© 2022


  • base editor
  • cancer immunotherapy
  • gene therapy
  • hematopoietic stem cell
  • multiplex gene editing
  • sickle cell

PubMed: MeSH publication types

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


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