Recent Advancements in Reducing the Off-Target Effect of CRISPR-Cas9 Genome Editing.

IF 5.3 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Biologics : Targets & Therapy Pub Date : 2024-01-18 eCollection Date: 2024-01-01 DOI:10.2147/BTT.S429411

Misganaw Asmamaw Mengstie, Muluken Teshome Azezew, Tadesse Asmamaw Dejenie, Assefa Agegnehu Teshome, Fitalew Tadele Admasu, Awgichew Behaile Teklemariam, Anemut Tilahun Mulu, Melaku Mekonnen Agidew, Dagnew Getnet Adugna, Habtamu Geremew, Endeshaw Chekol Abebe

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Abstract

The CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)) and the associated protein (Cas9) system, a young but well-studied genome-editing tool, holds plausible solutions to a wide range of genetic disorders. The single-guide RNA (sgRNA) with a 20-base user-defined spacer sequence and the Cas9 endonuclease form the core of the CRISPR-Cas9 system. This sgRNA can direct the Cas9 nuclease to any genomic region that includes a protospacer adjacent motif (PAM) just downstream and matches the spacer sequence. The current challenge in the clinical applications of CRISPR-Cas9 genome-editing technology is the potential off-target effects that can cause DNA cleavage at the incorrect sites. Off-target genome editing confuses and diminishes the therapeutic potential of CRISPR-Cas9 in addition to potentially casting doubt on scientific findings regarding the activities of genes. In this review, we summarize the recent technological advancements in reducing the off-target effect of CRISPR-Cas9 genome editing.

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减少 CRISPR-Cas9 基因组编辑脱靶效应的最新进展。

CRISPR-Cas（Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)）和相关蛋白（Cas9）系统是一种年轻但已被充分研究的基因组编辑工具，可为多种遗传疾病提供可行的解决方案。带有 20 个碱基用户定义间隔序列的单导核糖核酸（sgRNA）和 Cas9 内切酶构成了 CRISPR-Cas9 系统的核心。这种 sgRNA 可将 Cas9 核酸酶导向任何包含原间隔邻接基序（PAM）且正好位于间隔序列下游并与间隔序列相匹配的基因组区域。目前，CRISPR-Cas9 基因组编辑技术在临床应用中面临的挑战是潜在的脱靶效应，它可能导致 DNA 在不正确的位点被裂解。脱靶基因组编辑会混淆和削弱 CRISPR-Cas9 的治疗潜力，还可能对有关基因活性的科学发现产生怀疑。在这篇综述中，我们总结了最近在减少 CRISPR-Cas9 基因组编辑的脱靶效应方面取得的技术进展。

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