多能干细胞治疗视网膜退行性疾病:从实验室到临床转化的现状与挑战  

Pluripotent stem cells in the treatment of retinal degenerative diseases:Current situation and challenges from bench to bedside

作　　者：郭艳 鲍莉 纪猛 吴月红 GUO Yan;BAO Li;JI Meng;WU Yuehong(College of Life Science and Medicine,Zhejiang Sci-Tech University,Hangzhou 310018,China;Asia Stem Cell Therapeutics Co.,Ltd.,Hangzhou 310018,China)

机构地区：[1]浙江理工大学生命科学与医药学院,杭州310018 [2]浙江泉生生物工程有限公司,杭州310018

出　　处：《浙江理工大学学报（自然科学版）》2024年第1期130-144,共15页Journal of Zhejiang Sci-Tech University(Natural Sciences)

基　　金：企业委托研发项目(21040400-J)。

摘　　要：视网膜色素上皮细胞(Retinal pigment epithelial cells,RPE)是位于视网膜底部致密的细胞层,其损伤导致年龄相关性黄斑变性(Age-related macular degeneration,AMD)、Stargardt病(Stargardt macular dystrophy,STGD)和色素性视网膜炎(Retinitis pigmentosa,RP)等视网膜疾病,RPE移植已成为治疗RPE损伤性疾病的有效方案。来源于人多能干细胞(Human pluripotent stem cells,hPSC)的视网膜色素上皮细胞,具有与人原代RPE相似的功能和容易制备等优点,已成为RPE移植的最主要细胞来源之一。文章对hPSC-RPE治疗视网膜退行性疾病的临床试验进展进行了总结和归纳,并阐述了目前面临的挑战与风险。Retinal pigment epithelial(RPE)cells are located in a dense layer of cells at the bottom of the retina,and their damage can result in age-related macular degeneration(AMD),Stargardt macular dystrophy(STGD),Retinitis pigmentosa(RP)and other retinal degenerative diseases.RPE transplantation has become an effective treatment for such diseases at present.RPE cells derived from human pluripotent stem cells(hPSC),namely hPSC-RPE,have become the main source of RPE transplantation because of their similar function and easy preparation to human primary RPE.This article summarizes the clinical trials of hPSC-RPE in the treatment of retinal degenerative diseases,and expounds the challenges and risks we are facing at present.

关 键 词：人多能干细胞 视网膜色素上皮细胞 年龄相关性黄斑变性 STARGARDT病 色素性视网膜炎 细胞治疗 

分 类 号：Q291[生物学—细胞生物学]