基因编辑技术的演变及应用  

Evolution and application of gene editing technology

作　　者：马梦遥 张欣欣 刘永林 蔡雷[1] 贺林[1] Ma Mengyao;Zhang Xinxin;Liu Yonglin;Cai Lei;He Lin(Bio-X Institutes,Key Laboratory for the Genetics of Developmental and Neuropsychiatric Disorders(Ministry of Education),Shanghai Jiaotong University,Shanghai 200230,China;Tongji Hospital Affiliated to Tongji Medical College,Huazhong University of Science and Technology,Wuhan 430030,China;Sanya Maternal and Child Health Hospital,Sanya 572019,China)

机构地区：[1]上海交通大学Bio-X研究院遗传发育与精神神经疾病教育部重点实验室,上海200030 [2]华中科技大学同济医学院附属同济医院,武汉430030 [3]上海儿童医学中心三亚妇女儿童医院,三亚572019

出　　处：《国际遗传学杂志》2022年第3期176-184,共9页International Journal of Genetics

基　　金：中国科学技术部国家重点研发计划重点专项(2017YFC1001302);上海市科学技术委员会自然科学基金(19ZR1427700);上海精神卫生中心(19-K02)重性精神病重点实验室开放课题项目(13dz2260500)。

摘　　要：基因编辑技术是以改变靶基因序列为目的,实现定点突变、插入或敲除的技术。作为生命科学的新兴研究领域,基因编辑技术的不断发展与完善为基因的功能研究、基因检测和治疗提供了强有力的工具,极大地推动了生命科学的发展。本文首先介绍了基因编辑技术发展过程中的三种主要技术—锌指核酸酶(zinc-finger nucleases,ZFNs),类转录激活因子效应核酸酶(transcription activator-like effector nucleases,TALENs)和CRISPR-Cas9。通过改造现有Cas9核酸酶以及寻找新的具有特异性识别和切割目的序列的蛋白,适用范围更广泛的基因编辑系统被开发出来,推动了基因编辑技术的发展。本文同时对基因编辑技术的应用和存在的机遇及挑战进行了探讨,以增进对该技术及其未来研究的整体认识。Gene editing technology is a technology that aims to change the sequence of target genes to achieve targeted mutation,insertion or knockout.As an emerging research field in the life sciences,the continuous development and improvement of gene editing technology provides a powerful tool for the functional research,gene detection and treatment of genes,which has greatly promoted the development of life sciences.This article first introduces the three main technologies in the development of gene editing technology—zinc-finger nucleases(ZFNs),transcription activator-like effector nucleases(TALENs)and CRISPR-Cas9.Through the modification of existing Cas9 nucleases and the search for new proteins with specific identification and cleavage sequences of interest,gene editing systems with a wider range of applications have been developed,driving the development of gene editing technologies.This paper also discusses the application and existing opportunities and challenges of gene editing technology to enhance the overall understanding of the technology and its future research.

关 键 词：基因编辑 CRISPR ZFN TALEN Cas核酸酶 

分 类 号：Q789[生物学—分子生物学]