人尿源性诱导多能干细胞在遗传病治疗中的应用前景

Application prospect of human urine-drived induced pluripotent stem cells in treatment of genetic diseases

摘要:

遗传病传统的临床治疗方法只能减轻或矫正患者的临床症状，不能改变患者携带的致病基因。揭示遗传病的发病机制，利用基因治疗改变细胞中的遗传物质是根治遗传病的有效途径。干细胞是一类具有自我更新能力和多向分化潜能特性的细胞，其中人尿源性诱导多能干细胞具有容易获得、易于培养增殖、重编程率高等优点，且可被诱导分化为多种细胞和器官。该文总结了近五年来人尿源性诱导多能干细胞的高效重编程方法，并整理其在心脏、肌营养不良、脊髓型肌萎缩等相关遗传病的疾病模型复制、机制探索及可行性治疗方面的应用，为遗传病的临床治疗研究提供更多的思路。

Abstract:

The traditional clinical treatments of genetic diseases can only alleviate or correct the clinical symptoms of patients, but not change the pathogenic genes carried by patients. To reveal the pathogenesis of genetic diseases, using gene therapy to change the hereditary substances in cells is an effective way to cure genetic diseases. Stem cells are a kind of cells with self-renewal ability and multi-directional differentiation potential. The human urine-drived induced pluripotent stem cells have the advantages of easy access, easy culture and proliferation, high reprogramming rate, and can be induced to differentiate into a variety of cells and organs. This paper discussed the high efficient reprogramming methods of human urine-derived induced pluripotent stem cells in recent five years, and summarized their applications in the establishment of disease models, mechanism exploration and feasibility treatment of genetic diseases related to heart, muscular dystrophy, spinal muscular atrophy, etc., so as to provide more ideas for the clinical treatments and researches of genetic diseases.