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成簇规律间隔短回文重复序列(CRISPR)/CRISPR相关蛋白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].

作者信息

Sun S J, Huo J H, Geng Z J, Sun X Y, Fu X B

机构信息

College of Life Sciences, General Hospital of PLA, Beijing 100853, China.

出版信息

Zhonghua Shao Shang Za Zhi. 2018 Apr 20;34(4):253-256. doi: 10.3760/cma.j.issn.1009-2587.2018.04.013.

Abstract

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.

摘要

基因工程因其能够在基因组中精确定位疾病突变而备受全球关注。作为一种新的基因编辑技术,成簇规律间隔短回文重复序列(CRISPR)/CRISPR相关蛋白9(Cas9)系统操作简单、快速且能在特定基因位点精确操作。它克服了传统操作长期存在的问题。同时,干细胞是体外建立疾病模型的良好基础。因此,将干细胞与快速发展的基因操作技术相结合具有重要意义。在本综述中,我们主要聚焦于CRISPR/Cas9技术的机制及其在干细胞基因组编辑中的应用,以便为推动CRISPR/Cas9技术的快速应用和发展铺平道路。

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