CRISPR/Cas9基因组定点编辑技术在病毒性及遗传性疾病治疗中的应用及其进展  

Progress and Application in CRISPR/Cas9 on Treatment of Human Viral and Hereditary Diseases

作　　者：季彬[1,2] 仇建萍 周宋峰 陈莉[3] 朱远源[1,2,3] 王旻[1] 

机构地区：[1]中国药科大学生命科学与技术学院,南京210009 [2]百奥迈科生物技术有限公司,江苏南通226010 [3]南通大学病理教研室,江苏南通226019

出　　处：《抗感染药学》2017年第1期1-6,共6页Anti-infection Pharmacy

摘　　要：第3代基因组定点编辑技术(Clustered Regularly Interspaced Short Palindromic Repeat(CRISPR,成簇规律间隔短回文序列))介导的Cas9核酸酶系统,已被用于真核细胞的基因组编辑和基因修饰。CRISPR/Cas9技术有许多潜在的应用,包括对人类基因组进行编辑以治疗遗传缺陷疾病或基因突变疾病;删除病原体基因(如细菌基因组或病毒基因组),消灭病原体,达到消除传染性疾病的目的;也可用于农业生产(包括植物和动物的基因)中农作物的改造。综述基因组定点编辑技术CRISPR/Cas9在病毒感染性疾病和遗传缺陷性疾病中的应用文献,并对其研究进展作了分析。Clustered regularly interspaced short palindromic repeat（CRISPR） mediated Cas9 nucleases system is considered as the third generation of genome editing technology. CRISPR/Cas9 system has great potential for medical application, such as human genome editing for the treatment of the genetic deficiency disease or disease caused by gene mutations; altering the genome of the pathogens including bacterial genomes or viral genomes so as to eliminate the infectious disease. It can also be used for agriculture production, including plant and animal genetic modification. The literature of application of CRISPR/Cas9 system in human viral infection diseases and hereditary diseases has been reviewed, and its research progress is analyzed.

关 键 词：基因组定点编辑技术 CRISPR/Cas9 成簇规律间隔短回文序列 sgRNA 基因编辑 基因修饰 

分 类 号：R969.3[医药卫生—药理学]