CRISPR/Cas9系统的分子机制及其在人类疾病基因治疗中的应用

doi:10.16288/j.yczz.15-109

遗传 ›› 2015, Vol. 37 ›› Issue (10): 974-982.doi: 10.16288/j.yczz.15-109

CRISPR/Cas9系统的分子机制及其在人类疾病基因治疗中的应用

璩良, 李华善, 姜运涵, 董春升

苏州大学生物医学研究院，苏州大学医学部，苏州 215123

收稿日期:2015-03-16 出版日期:2015-10-20 发布日期:2015-10-20
通讯作者: 董春升，副教授，研究方向：免疫学。 E-mail：chunshengdong@suda.edu.cn
作者简介:璩良，本科生，专业方向：生物技术(免疫工程)。E-mail: quliangsuda@163.com
基金资助:
国家自然科学基金项目(编号：K113415011)，江苏省大学生创新创业训练计划项目(编号：201310285046Z)和苏州市科技局项目(编号：SYS201452)资助

The molecular mechanism of CRISPR/Cas9 system and its application in gene therapy of human diseases

Liang Qu， Huashan Li， Yunhan Jiang， Chunsheng Dong

Institutes of Biology and Medical Sciences (IBMS ), School of Medicine, Soochow University, Suzhou 215123, China

Received:2015-03-16 Online:2015-10-20 Published:2015-10-20

摘要/Abstract

摘要： CRISPR/Cas系统是广泛存在于细菌和古生菌中的适应性免疫系统，用来抵抗外来病毒或质粒的入侵。近几年，由Ⅱ型CRISPR/Cas适应性免疫系统改造而来的CRISPR/ Cas9基因组编辑技术蓬勃发展，被广泛地应用于生命科学研究的各个领域，并取得了革命性的变化。文章主要综述了CRISPR/Cas9基因组编辑技术的起源与发展及在生命科学各研究领域的应用，重点介绍了该系统在人类疾病基因治疗方面的最新应用及脱靶效应，以期为相关领域的科研人员提供参考。

关键词: CRISPR/Cas9系统, 适应性免疫, 基因组编辑

Abstract: ?CRISPR/Cas system is an adaptive immune system that confers resistance to exogenous virus or plasmid in bacteria and archaea. In recent years, the booming CRISPR/Cas9 genome editing technology modified from typeⅡ CRISPR/Cas adaptive immune system has been widely applied to various research fields of life science and led to revolutionary changes. In this review, we summarize the origin and development of CRISPR/Cas9 genome editing technology as well as its applications in life science research. We focus on the latest application of this system in gene therapy of human diseases and the associated side/off-target effects, which may provide references for researchers in related areas.

Key words: CRISPR/Cas9 system, adaptive immune system, genome editing

璩良, 李华善, 姜运涵, 董春升. CRISPR/Cas9系统的分子机制及其在人类疾病基因治疗中的应用[J]. 遗传, 2015, 37(10): 974-982.

Liang Qu， Huashan Li， Yunhan Jiang， Chunsheng Dong. The molecular mechanism of CRISPR/Cas9 system and its application in gene therapy of human diseases[J]. HEREDITAS(Beijing), 2015, 37(10): 974-982.

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CRISPR/Cas9系统的分子机制及其在人类疾病基因治疗中的应用

The molecular mechanism of CRISPR/Cas9 system and its application in gene therapy of human diseases

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