Off-target effects in CRISPR/Cas9 gene editing

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 t...

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Bibliographic Details
Published inFrontiers in bioengineering and biotechnology Vol. 11; p. 1143157
Main Authors Guo, Congting, Ma, Xiaoteng, Gao, Fei, Guo, Yuxuan
Format Journal Article
LanguageEnglish
Published Switzerland Frontiers Media S.A 09.03.2023
Subjects
Bioengineering and Biotechnology
Cas9/sgRNA complex
CRISPR/Cas9
gene editing
gene therapy
off-target effects
gene therapy
CRISPR/Cas9
off-target effects
gene editing
Cas9/sgRNA complex
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Summary: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.
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Xiaoping Bao, Purdue University, United States
This article was submitted to Preclinical Cell and Gene Therapy, a section of the journal Frontiers in Bioengineering and Biotechnology
Xiao-Bing Zhang, Institute of hematology, China
Edited by: Kai Wang, Peking University, China
Reviewed by: Nathan VanDusen, Indiana University Bloomington, United States
ISSN:2296-4185
2296-4185
DOI:10.3389/fbioe.2023.1143157