Off-target effects in CRISPR/Cas9 gene editing
- PMID: 36970624
- PMCID: PMC10034092
- DOI: 10.3389/fbioe.2023.1143157
Off-target effects in CRISPR/Cas9 gene editing
Abstract
Gene editing stands for the methods to precisely make changes to a specific nucleic acid sequence. With the recent development of the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system, gene editing has become efficient, convenient and programmable, leading to promising translational studies and clinical trials for both genetic and non-genetic diseases. A major concern in the applications of the CRISPR/Cas9 system is about its off-target effects, namely the deposition of unexpected, unwanted, or even adverse alterations to the genome. To date, many methods have been developed to nominate or detect the off-target sites of CRISPR/Cas9, which laid the basis for the successful upgrades of CRISPR/Cas9 derivatives with enhanced precision. In this review, we summarize these technological advancements and discuss about the current challenges in the management of off-target effects for future gene therapy.
Keywords: CRISPR/Cas9; Cas9/sgRNA complex; gene editing; gene therapy; off-target effects.
Copyright © 2023 Guo, Ma, Gao and Guo.
Conflict of interest statement
The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. The handling editor KW declared a shared affiliation with the author YG at the time of review.
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