基因组定点编辑技术研究进展.pdfVIP

第25卷第7期 生命科学 Vol. 25, No. 7 2013年7月 Chinese Bulletin of Life Sciences Jul., 2013 文章编号：1004-0374(2013)07-0735-08 ∙ 技术与应用∙ 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：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 中图分类号：Q-33 ；Q78 A 文献标志码 ： 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: 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/Ca