CRISPR系统转化医学研究进展  

Research Progress of CRISPR System in Translational Medicine

作　　者：叶洲杰 王心睿[2,3,4] YE Zhou-jie;WANG Xin-rui(Fujian Children’s Hospital,Fuzhou 350011;Medical Research Center,Fujian Maternity and Child Health Hospital,Fuzhou 350001;Fujian Key Laboratory of Women and Children’s Critical Diseases Research,Fujian Maternity and Child Health Hospital,Fuzhou 350001;Key Laboratory of Technical Evaluation of Fertility Regulation for Non human Primate,National Health and Family Planning Commission,Fuzhou 350013)

机构地区：[1]福建省儿童医院,福州350011 [2]福建医科大学附属福建省妇幼保健院医学研究中心,福州350001 [3]福建省妇幼保健院福建省妇儿重大疾病研究重点实验室,福州350001 [4]国家卫健委非人灵长类生育调节技术评价重点实验室,福州350013

出　　处：《生物技术通报》2020年第11期188-197,共10页Biotechnology Bulletin

基　　金：福建省妇幼保健院科研基金项目(YCXB 18-05,YCXM 19-03)。

摘　　要：CRISPR系统作为在现今科学研究中对基因编辑运用广泛的一项技术,在后基因组学时代对于基因功能研究、疾病发生发展机制以及基因靶向药物等研究具有重要意义。目前人们运用CRISPR/Cas9编辑技术成功构建细胞和模式生物模型用于临床医学研究。CRISPR/Cas9作为一类强大的基因编辑技术不单只运用于基因功能缺失或外源基因敲入等研究中,CRISPR基因敲除文库筛选系统、CRISPR/dCas9基因激活系统、CRISPR干扰技术和CRISPR分子诊断技术等CRISPR转化医学研究将CRISPR应用于基因高通量筛选、基因沉默导致疾病发生、跨表观遗传修饰激活基因表达、可逆性干扰靶标基因表达以及运用CRISPR分子诊断检测病原微生物感染性疾病等临床研究中,推动生命科学和临床医学等多学科的发展。针对CRISPR系统在转化医学中的研究应用进行阐述。CRISPR system is the most widely used technology for gene editing in the current scientific researches.In the post genomic era,CRISPR system is of great significance for gene function research,disease development mechanism and gene targeted drug research.At present,CRISPR/Cas9 editing technology has been used to successfully construct cell and biological models for clinical medical research.However,CRISPR/Cas9,as a powerful gene editing technology,is not only used for gene deletion or gene knock-in,but also applied in the gene high-throughput screening,research on disease caused by gene silencing,cross epigenetic modification activating gene expression,reversible interference of target gene expression and molecular diagnosis of pathogenic microorganism infectious diseases via the research and application of CRISPR/Cas9 in translational medicine in recent years,such as the screening system of CRISPR gene-knockout library,CRISPR/dCas9 gene activation system,CRISPR interference technology and CRISPR molecular diagnosis technology,etc.,which promotes the application and development of CRISPR in translational medicine research.

关 键 词：CRISPR/Cas9 CRISPR诊断 CRISPR基因敲除文库 CRISPR激活系统 CRISPR干扰 

分 类 号：Q789[生物学—分子生物学]