CRISPR/Cas9编辑引起的非预期大靶基因修饰的检测和定量

IF 4.7 3区 工程技术 Q2 ENGINEERING, BIOMEDICAL Current Opinion in Biomedical Engineering Pub Date : 2023-06-16 DOI:10.1016/j.cobme.2023.100478
So Hyun Park, Mingming Cao, Gang Bao
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引用次数: 2

摘要

基于CRISPR/Cas9的基因编辑通常通过在细胞中的预定目标位点上创建DNA双链断裂(DSB)来发挥作用。最近的报道显示,除了小的插入和缺失外,CRISPR/ cas9诱导的DSB还发生了意想不到的靶向大基因修饰,包括大的缺失、插入和染色体重排。这些靶向大基因修饰的频率很高,无法通过基于标准短程PCR的检测检测到,从而导致数据误解,降低疗效,并在治疗性基因编辑中存在潜在的安全性问题。在这里,我们总结了分析大型靶向基因编辑结果及其对临床应用的影响的最新进展,并讨论了未来改进的机会。
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Detection and quantification of unintended large on-target gene modifications due to CRISPR/Cas9 editing

CRISPR/Cas9 based gene editing typically functions by creating a DNA double-strand break (DSB) at the intended target locus in a cell. Recent reports showed the occurrence of unintended on-target large gene modifications by CRISPR/Cas9-induced DSB, including large deletions, insertions, and chromosomal rearrangements, in addition to small insertions and deletions. These on-target large gene modifications can have high frequencies, undetectable by standard short-range PCR based assays, leading to data misinterpretation, reduced efficacy, and potential safety concerns in therapeutic gene editing. Here, we summarize the recent advances in analyzing large on-target gene editing outcomes and their implications to clinical application, and discuss opportunities for future improvements.

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来源期刊
Current Opinion in Biomedical Engineering
Current Opinion in Biomedical Engineering Medicine-Medicine (miscellaneous)
CiteScore
8.60
自引率
2.60%
发文量
59
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