《生命科学》 2013, 25(7): 735-741

基因组定点编辑技术的研究进展

张智辉，董少忠^*，寸　韡^*

(中国医学科学院&北京协和医学院医学生物学研究所，云南省重大传染病疫苗研发重点实验室，昆明 650118)

摘　要：摘　要：基因组编辑是建立在基因靶向修饰的基础上，对生物基因组进行改造的一项新技术。通过利用人工核酸酶ZFn、TALEN 和细菌获得性免疫系统CRISPR，可在靶位点制造 DNA 双链切口进而诱导细胞内源性修复机制，通过同源重组修复或非同源末端连接途径实现基因敲除、替换和纠正。对目前3 个主要的基因组定点编辑技术的应用和发展作一综述。
    关键词：定点基因组编辑；锌指核酸酶；TALEN ；CRISPR/Cas9

Progress in targeted genome editing technologies

ZHANG Zhi-Hui， DONG Shao-Zhong^*， CUN Wei^*

(Institute of Medical Biology， Chinese Academy of Medical Sciences & Peking Union Medical College， Yunnan Key Laboratory of Vaccine Research & Development on Severe Infectious Disease， Kunming 650118， China)

Abstract: Abstract: Genome editing is a novel technology based on precise genome-targeting modification to enable systematic reverse engineering of causal genetic variation. A set of gene editing techniques， zinc finger nucleases，transcription activator-like effector nucleases and bacterial CRISPR (clustered regularly interspaced short palindromic repeats)/Cas adaptive immunity system， have been used to cleave the double-strand DNA at specific site， followed by endogenous DNA repair. These techniques， therefore， make precise changes to the DNA of living cell by， for example， knocking out a gene， correcting a genetic mutation or replacing DNA fragment at the specific site. The new progress in targeted genome editing technologies was discussed in this paper.
    Key words: targeted genome editing; zinc finger nucleases; TALEN; CRISPR/Cas9