Clustered Regularly Interspaced Short Palindromic Repeats and Clustered Regularly Interspaced Short Palindromic Repeats-Associated Protein 9 System: Factors Affecting Precision Gene Editing Efficiency and Optimization Strategies.

IF 3.9 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Human gene therapy Pub Date : 2023-12-01 Epub Date: 2023-10-17 DOI:10.1089/hum.2023.115
Jiawen Li, Chuxi Tang, Guozheng Liang, Huiqun Tian, Guanxi Lai, Yixiang Wu, Shiwen Liu, Wenfeng Zhang, Song Liu, Hongwei Shao
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Abstract

The clustered regularly interspaced short palindromic repeat (CRISPR)-CRISPR-associated (Cas) system is a powerful genomic DNA editing tool. The increased applications of gene editing tools, including the CRISPR-Cas system, have contributed to recent advances in biological fields, such as genetic disease therapy, disease-associated gene screening and detection, and cancer therapy. However, the major limiting factor for the wide application of gene editing tools is gene editing efficiency. This review summarizes the recent advances in factors affecting the gene editing efficiency of the CRISPR-Cas9 system and the CRISPR-Cas9 system optimization strategies. The homology-directed repair efficiency-related signal pathways and the form and delivery method of the CRISPR-Cas9 system are the major factors that influence the repair efficiency of gene editing tools. Based on these influencing factors, several strategies have been developed to improve the repair efficiency of gene editing tools. This review provides novel insights for improving the repair efficiency of the CRISPR-Cas9 gene editing system, which may enable the development and improvement of gene editing tools.

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簇状规则间隔短回文重复序列和簇状规则间隙短回文反复序列相关蛋白9系统:影响精确基因编辑效率的因素和优化策略。
集群规则间隔短回文重复序列(CRISPR)-CRISPR相关(Cas)系统是一种强大的基因组DNA编辑工具。包括CRISPR-Cas系统在内的基因编辑工具应用的增加,促进了生物领域的最新进展,如遗传疾病治疗、疾病相关基因筛查和检测以及癌症治疗。然而,基因编辑工具广泛应用的主要限制因素是基因编辑效率。本文综述了影响CRISPR-Cas9系统基因编辑效率的因素和CRISPR-Cas9系统优化策略的最新进展。同源性定向修复效率相关信号通路以及CRISPR-Cas9系统的形式和递送方法是影响基因编辑工具修复效率的主要因素。基于这些影响因素,已经制定了几种策略来提高基因编辑工具的修复效率。这篇综述为提高CRISPR-Cas9基因编辑系统的修复效率提供了新的见解,这可能有助于基因编辑工具的开发和改进。
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来源期刊
Human gene therapy
Human gene therapy 医学-生物工程与应用微生物
CiteScore
6.50
自引率
4.80%
发文量
131
审稿时长
4-8 weeks
期刊介绍: Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
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