《生命科学》 2019, 31(7): 723-730

CRISPR/Cas9基因编辑技术在糖尿病细胞治疗中的应用研究进展

王晗月1，2，杨晓菲1，胡巢凤2*，李富荣1，2*

(1 暨南大学第二临床医学院(深圳市人民医院)转化医学协同创新中心，深圳 518020；2 暨南大学基础医学院病理生理学系，广州 510632)

摘　要：摘　要：近几年来，基因编辑技术快速发展，为在特定位置精确操控基因组提供了一个有效而精确的工具。CRISPR/Cas9 系统是一种新型的基因编辑工具，可实现对人类基因组的高精度修饰，是疾病建模和治疗的可行选择。随着CRISPR/Cas9 系统技术的广泛应用，在糖尿病领域有不少相关研究出现。该文从干细胞分化为β 细胞、基因编辑重编程为β 细胞以及修饰基因三个方面总结了CRISPR/Cas9 技术在糖尿病治疗中的应用。

CRISPR/Cas9 gene editing in diabetes cell therapy: recent advances

WANG Han-Yue1，2， YANG Xiao-Fei1， HU Chao-Feng2*， LI Fu-Rong1，2*

(1 Translational Medicine Collaborative Innovation Center， The Second Clinical Medical College (Shenzhen People’s Hospital) of Jinan University， Shenzhen 518020， China; 2 Department of Pathology and Pathophysiology， Jinan University， Guangzhou 510632， China)

Abstract: Abstract: In recent years， the rapid development of gene editing technology has provided an effective and accurate tool for the manipulation of genomes at specific locations. CRISPR/Cas9 system is a new gene editing tool， which can achieve high-precision modification on human genome and is a feasible strategy for disease modeling and treatment. CRISPR/Cas9 system has been widely applied in many research fields， including diabetes cell therapy. In this review， we summarized recent advances of CRISPR/Cas9 in the treatment of diabetes mellitus， especially in the differentiation of stem cells into β cells， direct reprogramming into beta cells， and genes modification.