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

作　　者：袁艳萍 郑志娟[1] 李运伦[2] Yan-ping Yuan;Zhi-juan Zheng;Yun-lun Li(Shandong University of Traditional Chinese Medicine,Jinan,Shandong 250355,China;Affiliated Hospital of Shandong University of Traditional Chinese Medicine,Jinan,Shandong 250014,China)

机构地区：[1]山东中医药大学,山东济南250355 [2]山东中医药大学附属医院,山东济南250014

出　　处：《中国现代医学杂志》2022年第3期38-43,共6页China Journal of Modern Medicine

基　　金：山东省泰山学者工程专项经费资助(No:ts201712042);山东省医药卫生科技发展计划项目(No:2019WS558)。

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。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.

关 键 词：遗传病 人尿源性诱导多能干细胞 尿液细胞重编程 机制研究 可行性治疗 

分 类 号：R596[医药卫生—内科学]