CRISPR系统及其在遗传病治疗中应用的研究进展  

The progress of CRISPR system and its application in treatment of hereditary diseases

作　　者：白健[1] 曹乾[1] 王亮[1] 韩骅[1] 秦鸿雁[1] 

机构地区：[1]第四军医大学基础部医学遗传与发育生物学教研室,西安710032

出　　处：《国际遗传学杂志》2017年第1期45-50,共6页International Journal of Genetics

基　　金：国家自然科学基金（81530018）

摘　　要：CRISPR （clustered regularly interspaced short palindromic repeats ）系统是细菌和古生菌用于抵抗外源性病毒和质粒入侵的适应性免疫防御系统，近年来遗传工程迅猛发展，CRISPR系统中的Cas9、Cpfl等多种蛋白已被改造为基因组编辑的关键工具。对于由遗传物质发生改变而引起的或者由致病基因突变所控制的遗传病，CRISPR系统已被证实是编辑修正致病基因的有力手段，并有望成为人类遗传病治疗的新策略。本文主要从CRISPR系统的研究进展及其在遗传病治疗中的应用进行综述。CRISPR system is an adaptive immune defense system in bacteria and archaea, which functions to resist exogenous viruses and plasmids. Recent years have witnessed the great development in genetics engineering, especially Cas9, Cpfl and other proteins in CRISPR system, and recognized as vital tools in genome editing. In regard to hereditary diseases caused by genetic material changing or pathogenic gene mutation, CRISPR system has been proved as a powerful tool to edit and correct pathogenic genes, and a promising strategy to cure human hereditary diseases. In this review, we mainly discuss the progress of CRISPR system and its application in hereditary diseases therapy.

关 键 词：CRISPR系统 基因组编辑 遗传病 

分 类 号：Q78[生物学—分子生物学] R596[医药卫生—内科学]