The molecular mechanism of CRISPR/Cas9 system and its application in gene therapy of human diseases.
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 type2 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.
Institutes of Biology and Medical Sciences (IBMS), School of Medicine, Soochow University, Suzhou 215123, China.
Liang
L
Qu
Institutes of Biology and Medical Sciences (IBMS), School of Medicine, Soochow University, Suzhou 215123, China.
Hua-shan
HS
Li
Institutes of Biology and Medical Sciences (IBMS), School of Medicine, Soochow University, Suzhou 215123, China.
Yun-han
YH
Jiang
Institutes of Biology and Medical Sciences (IBMS), School of Medicine, Soochow University, Suzhou 215123, China.
Chun-sheng
CS
Dong
D056704:Adaptive Immunity / Q000235:genetics*; D064113:CRISPR-Cas Systems; D004720:Endonucleases / Q000235:genetics / Q000378:metabolism; D005818:Genetic Engineering / Q000379:methods* / Q000639:trends; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D015658:HIV Infections / Q000235:genetics / Q000276:immunology / Q000628:therapy; D006801:Humans; D008957:Models, Genetic; D009369:Neoplasms / Q000235:genetics / Q000276:immunology / Q000628:therapy; D000094704:RNA, Guide, CRISPR-Cas Systems / Q000235:genetics / Q000378:metabolism
D000094704:RNA, Guide, CRISPR-Cas Systems; D004720:Endonucleases