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

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

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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

Abstract

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

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

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