Off-target effects in CRISPR/Cas9 gene editing.
Front Bioeng Biotechnol, 2023;11:1143157.
Guo C[1, 2], Ma X[3], Gao F[3], Guo Y[1, 2, 4, 5]
Affiliations
PMID: 36970624DOI: 10.3389/fbioe.2023.1143157
Impact factor: 6.064
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
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