碱基编辑系统的研究进展  

Advances in base editing systems

作　　者：钟静丽 林健香 周建奎 乔云波 ZHONG Jingli;LIN Jianxiang;ZHOU Jiankui;QIAO Yunbo(School of Life Sciences,Guangzhou University,Guangzhou 510006,Guangdong,China;Shanghai Institute of Precision Medicine,Ninth People’s Hospital,Shanghai Jiao Tong University School of Medicine,Shanghai 200125,China)

机构地区：[1]广州大学生命科学学院,广东广州510006 [2]上海交通大学医学院附属第九人民医院上海精准医学研究院,上海200125

出　　处：《生物工程学报》2024年第5期1271-1292,共22页Chinese Journal of Biotechnology

基　　金：国家自然科学基金(82371668);上海市“科技创新行动计划”自然科学基金(23ZR1437700);上海市科委合成生物学重点专项(23HC1400700)。

摘　　要：基于可编程核酸酶的基因编辑工具表现出编辑高效、产物纯度高、编辑副产物少的优势,已广泛应用于生物医药研发和作物育种。鉴于研究和应用的需求多样化,开发功能特异的碱基编辑器成为基因编辑领域的研究热点。目前由规律成簇的间隔短回文重复序列系统及相关蛋白(clustered regularly interspaced short palindromic repeats and CRISPR-associated, CRISPR-Cas)和转录激活因子类效应因子(transcription activator-like effector, TALE)系统衍生的基因编辑系统包括单碱基编辑器、双碱基编辑器、线粒体碱基编辑器和CRISPR相关转座酶系统等。本文全面梳理了碱基编辑系统的发展历程,总结了各类碱基编辑器的特点、脱靶效应、优化和改良策略等,为基因编辑系统的进一步改进和应用提供参考。Programmable nucleases-based genome editing systems offer several advantages,such as high editing efficiency,high product purity,and fewer editing by-products.They have been widely used in biopharmaceutical research and crop engineering.Given the diverse needs for research and application,developing functional base editors has become a major focus in the field of genome editing.Currently,genome editing systems derived from clustered regularly interspaced short palindromic repeats and CRISPR-associated(CRISPR-Cas)and transcription activator-like effector(TALE)systems include single base editors,dual base editors,mitochondrial base editors,and CRISPR-related transposase systems.This review provides a comprehensive overview of the development of base editing systems,summarizes the characteristics,off-target effects,optimization,and improvement strategies of various base editors,and provides insights for further improvement and application of genome editing systems.

关 键 词：规律成簇的间隔短回文重复序列系统及相关蛋白9(CRISPR-Cas9) 碱基编辑器 转录激活因子类效应因子(TALE)系统 脱氨酶 

分 类 号：Q78[生物学—分子生物学]