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Updated: Aug 14, 2025

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
Published on: May 25, 2018
CRISPR-Cas9 base editors and their current role in human therapeutics
Walker S Lahr1, Christopher J Sipe1, Joseph G Skeate1
1Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA; Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, USA; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA; Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA; Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA.
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.
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