成簇规律间隔短回文重复序列及其相关蛋白基因9系统在干细胞研究中的应用进展  

Advances in application of clustered regularly interspaced short palindromic repeats （CRISPR）/CRISPR-associated 9 system in stem cells research

作　　者：孙苏静 霍嘉慧 耿志军[3] 孙晓艳[1] 付小兵[1] Sun Sujing;Huo Jiahui;Geng Zhijun;Sun Xiaoyan;Fu Xiaobing(College of Life Sciences, General Hospital of PLA, Beijing 100853, China)

机构地区：[1]解放军总医院生命科学院,北京100853 [2]天津医科大学研究生院 [3]解放军总医院第一附属医院全军创伤修复与组织再生重点实验室暨皮肤损伤修复与组织再生北京市重点实验室

出　　处：《中华烧伤杂志》2018年第4期253-256,共4页Chinese Journal of Burns

基　　金：国家自然科学基金（81121004、81230041、81421064、81372067）

摘　　要：随着对DNA碱基精确识别的核酸酶研究的深入，第3代基因编辑技术，如锌指核酸酶（ZFN）技术、类转录激活因子样效应物核酸酶（TALEN）技术等发展迅速，由于该类技术能够精确地进行DNA水平的特定位点敲除、插入及替换，加深研究者对生物基因组变化的了解，从而进一步探讨调控生物表型的内源性基因位点，最大限度在体外细胞模型中模拟疾病状态，因此越来越受到研究者的重视。这些技术在真核细胞中的应用也被证实是简便有效且具有位点特异性的，目前已成为实验室的一种常规基因编辑手段。虽然这些技术已进入临床应用阶段，但其脱靶率高、细胞毒性大及基因修饰类型单一等不足也亟待解决。Gene engineering has attracted worldwide attention because of its ability of precise location of disease mutations in genome. As a new gene editing technology, clustered regularly interspaced short palindromic repeats （CRISPR）/CRISPR-associated 9 （Cas9） system is simple, fast, and accurate to operate at a specific gene site. It overcomes the long-standing problem of conventional operation. At the same time, stem cells are a good foundation for establishing disease model in vitro. Therefore, it has great significance to combine stem cells with the rapidly developing gene manipulation techniques. In this review, we mainly focus on the mechanism of CRISPR/Cas9 technology and its application in stem cell genomic editing, so as to pave the way for promoting rapid application and development of CRISPR/Cas9 technology.

关 键 词：干细胞 成簇规律间隔短回文重复序列及其相关蛋白基因9 基因编辑 

分 类 号：Q78[生物学—分子生物学] R329.2[医药卫生—人体解剖和组织胚胎学]