(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.

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