Wang Yang-Min, Wang Hao-Zheng, Jian Ying-Zhen, Luo Zhong-Tao, Shao Hong-Wei, Zhang Wen-Feng
Guang dong Province Key Laboratory of Biotechnology Drug Candidates, Guang dong Pharmaceutical University, Guang zhou, P.R. China.
School of Biosciences and Biopharmaceutics, Guang dong Pharmaceutical University, Guang zhou, P.R. China.
Hum Gene Ther. 2022 Apr;33(7-8):358-370. doi: 10.1089/hum.2021.283. Epub 2022 Mar 1.
The clustered regularly interspaced short palindromic repeats (CRISPR) system is inarguably the most valuable gene editing tool ever discovered. Currently, three classes of CRISPR-based genome editing systems have been developed for gene editing, including CRISPR/CRISPR associate system (Cas) nucleases, base editors, and prime editors. Ever-evolving CRISPR technology plays an important role in medicine; however, the biggest obstacle to its use in clinical practice is the induction of off-target effects (OTEs) during targeted editing. Therefore, continuous improvement and optimization of the CRISPR system for reduction of OTEs is a major focus in the field of CRISPR research. This review aims to provide a comprehensive guide for optimization of the CRISPR-based genome editing system.
成簇规律间隔短回文重复序列(CRISPR)系统无疑是迄今发现的最有价值的基因编辑工具。目前,已开发出三类基于CRISPR的基因组编辑系统用于基因编辑,包括CRISPR/CRISPR相关系统(Cas)核酸酶、碱基编辑器和引导编辑器。不断发展的CRISPR技术在医学中发挥着重要作用;然而,其在临床实践中应用的最大障碍是靶向编辑过程中会诱导脱靶效应(OTE)。因此,持续改进和优化CRISPR系统以减少脱靶效应是CRISPR研究领域的一个主要重点。本综述旨在为基于CRISPR的基因组编辑系统的优化提供全面指导。