CRISPR/Cas基因编辑技术及其在血液系统疾病中的应用

Research Progress on Application of CRISPR/Cas Genome Editing Technology in Hematological Diseases——Review

CRISPR/Cas基因编辑技术是近年来新兴的对特定位点进行基因编辑的技术,利用CRISPR/Cas系统在某一特定位点导入目的基因,可以使有基因缺陷的细胞恢复正常功能,也可以在正常细胞内导入疾病相关基因从而构建相应疾病模型。CRISPR/Cas技术具有精确的打靶作用,作为基因剪切和编辑工具而广泛应用于各种实验,能够帮助深入研究基因功能以及治疗遗传性疾病。目前,CRISPR/Cas技术正成为农业、畜牧业、生物技术及医学领域的研究热点,本文就CRISPR/Cas系统在血液系统疾病中的最新研究进展作一综述。

CRISPR/Cas genome editing technology is a newly developed powerful tool for genetic manipulation,which can be used to manipulate the genome at specific locations precisely,to restore the function of genetic defect cells,and to develop various disease models.In recentl years,with the advances of precise genome manipulation,CRISPR/Cas technology has been applied to many aspects of diseases research and becomes an unique tool to investigate gene function and discover new therapeutic targets for genetic diseases.Nowadays,CRISPR/Cas technology has been a hot research point in agriculture,graziery,biotechnology and medicine.This review focuses on the recent advances in CRISPR/Cas technology and its application in hematological diseases.