《生命科学》 2015, 27(1): 12-19

靶向核酸酶介导基因编辑技术的发展

项光海，王皓毅*

(中国科学院动物研究所计划生殖生物学国家重点实验室，北京 100190)

摘　要：摘　要：对目标基因组位点进行靶向修饰一直是基因工程研究的重点。靶向基因编辑技术能够有效地用于建立动物和细胞疾病模型、培育动植物新品种，并具有治疗遗传疾病的重大潜力。近年来靶向核酸酶技术取得了重大的进展，逐渐成为基因编辑的主流工具。综述了锌指核酸酶(ZFN)、类转录激活样效应因子核酸酶(TALEN)、规律成簇间隔短回文重复序列(CRISPR/Cas) 这三大靶向基因编辑系统的原理和研究进展，并讨论了其局限性和未来的发展方向。

Programmable site-specific DNA endonucleases: powerful tools for genome engineering

XIANG Guang-Hai， WANG Hao-Yi*

(State Key Laboratory of Reproductive Biology， Institute of Zoology，
    Chinese Academy of Sciences， Beijing 100190， China)

Abstract: Abstract: Genome engineering technologies are invaluable tools for understanding the function of genes in health and disease. In recent years， the programmable site-specific DNA endonucleases， including zinc finger nucleases (ZFNs)， transcription activator-like effector nucleases (TALENs) and the clustered， regularly interspaced short palindromic repeat (CRISPR) system， have gained tremendous popularity and become widely used for genome engineering in cell lines and animal species ranging from Drosophila to primates. Here， we reviewed the principle，progress， limitation and prospect of ZFN， TALEN and CRISPR/Cas systems for genome engineering.