CRISPR-Cas9基因编辑技术在病毒感染疾病治疗中的应用  

The application of CRISPR-Cas9 gene editing technology in viral infection diseases

作　　者：殷利眷 胡斯奇[1] 郭斐[1] 

机构地区：[1]中国医学科学院/北京协和医学院病原生物学研究所,卫生部病原系统生物学重点实验室,北京100730

出　　处：《遗传》2015年第5期412-418,共7页Hereditas（Beijing)

基　　金：国家自然科学基金项目(编号:81371808)资助

摘　　要：CRISPR-Cas9基因编辑技术是基于细菌或古细菌CRISPR介导的获得性免疫系统衍生而来,由一段RNA通过碱基互补配对识别DNA,指导Cas9核酸酶切割识别的双链DNA,诱发同源重组或非同源末端链接,进而实现在目的 DNA上进行编辑。病毒通过特异的受体侵染细胞,其基因组在细胞内发生复制、转录、翻译等过程完成其生活周期,某些DNA病毒或逆转录病毒基因组会整合到宿主基因组中。基因治疗是病毒感染疾病治疗的新趋势。因此,基因编辑技术在持续感染的病毒或潜伏感染病毒疾病治疗中具有重大的潜在意义。文章主要从CRISPR-Cas9作用机制以及在病毒感染疾病治疗中的应用等方面进行了综述。The RNA-guided Cas9 nuclease from microbial clustered regularly interspaced short palindromic re- peats （CRISPR） adaptive immune system has been used to facilitate efficient genome engineering in eukaryotic cells. The specific targeted genome is recognized and cut by gRNA-directed CRISPR/Cas9 complex, specifically by the endonuclease Cas9. The targeted gene locus could be repaired either by homology-directed repair or nonhomologous end joining, thus achieving a desired editing outcome. Viruses infect ceils through specific receptors, and then the viral genome is transcribed, replicated and translated to complete its life cycle. As a result, some DNA virus and re- trovirus genomes are integrated into the cellular genome. Gene therapy is a new trend to treat viral infected diseases. Given its designable sequence-specific editing of the targeted genome, CRISPR/Cas9 has tremendous potential in treating persistent and latent viral infections. In this review, we summarize the mechanism and progresses of CRISPR/Cas9, and also highlight its therapeutic application in infectious diseases.

关 键 词：CRISPR 同源重组 非同源末端链接 基因编辑 病毒 

分 类 号：R511[医药卫生—内科学]