基于诱导多能干细胞的基因编辑和细胞治疗

Gene Editing and Cell Therapy Based on Induced Pluripotent Stem Cells

诱导多能干细胞(induced pluripotent stem cells,i PSCs)是指分化细胞重编程恢复到类似胚胎干细胞,具有自我更新、多向分化等潜能的一类细胞。基因编辑是对基因组特定位点进行的遗传操作,可方便快捷地实现靶向遗传修饰。随着重编程效率提高和安全性等的完善,对患者来源的i PSCs进行基因编辑、校正致病突变并用于细胞治疗正成为转化医学研究的热点。该文在简单介绍基因编辑原理和方法的基础上,重点综述了近年来基于i PSCs的基因编辑和细胞治疗的研究进展,对存在的问题进行了讨论,并对其在再生医学领域的发展前景进行了展望。

Induced pluripotent stem cells （iPSCs） are embryonic stem cell-like cells with self-renewal and differentiation capacity, which are reprogrammed from adult cells. Gene editing for the specific sites of genome can achieve the targeted genetic modification conveniently and efficiently. With improvement of efficacy and safety in cell reprogramming, gene editing and genetic correction for the pathogenic mutation of iPSCs derived from patients are attracting increasing attention on translational medicine, which open a remarkable avenue for cell therapy. In this review, we summarized the principles of gene editing, and then introduced the advance of gene editing and cell therapy in iPSCs. Occurring challenges and perspectives were also discussed.