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

Abstract

Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification. This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes. 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. 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.