CRISPR/Cas9系统在现代生物学研究和临床试验中的应用

 规律成簇间隔短回文重复结构(clustered regularly interspaced short palindromic repeats,CRISPR)/CRISPR相关蛋白9(CRISPR-associated protien 9,Cas9)系统在生物医学研究中被广泛应用于基因编辑及探索基因的功能。这种基因编辑技术越来越多地运用在人类疾病的治疗中,包括巴斯综合征、杜氏肌萎缩症、血友病、地中海贫血和囊性纤维化。CRISPR/Cas 9基因编辑系统可以在体外细胞实验和体内动物实验上修复突变的DNA序列,也能改变 CCR5、PD-1/L1、CAR-T等基因序列,用于控制 HIV病毒的入侵及提高肿瘤免疫治疗的疗效。该技术还被运用于诱导多能干细胞(induced pluripotent stem cells,iPS)分化,形成某些具有功能的器官用于器官移植。本文综述了CRISPR/Cas 9系统的来源、结构和作用原理,以及其在现代生物学研究和基因治疗领域的应用。

The application of CRISPR/Cas 9 system in biological research and clinical trial

Clustered regularly interspaced short palindromic repeats (CRISPR) is the clusters interval short palindrome repetition structure,CRISPR-associated protien 9 (Cas 9) is CRISPR related proteins.The CRISPR/Cas 9 system is widely used to edit gene in biomedical research and to explore the gene function.Recently,the gene editing techniques are wildly used in the human diseases therapy,including bath syndrome,duchenne muscular dystrophy,hemophilia,Mediterranean anemia and cystic fibrosis.CRISPR/Cas 9 system can repair DNA sequence in vitro and in vivo,it can also change the CCR5,PD-1/L1,CAR-T gene sequence.It also could be used to control the HIV virus invasion and improve the tumor immunotherapy.In addition,CRISPR/Cas 9 system has been used in induced pluripotent stem cells (iPS) evolving into certain functional organs,which were used to treat organ transplantation.This article summarized the source,structure and mechanism of CRISPR/Cas 9 system,and its application in the field of modern biology and gene therapy.