基于类器官的视网膜色素变性疾病模型构建及研究进展  

Construction and research progress of organoid models for retinitis pigmentosa

作　　者：谢林瑶(综述) 陈建苏[1] 郭永龙(审校) Xie Linyao;Chen Jiansu;Guo Yonglong(School of Basic Medicine and Public Health,Jinan University,Guangzhou 510632,China;College of Veterinary Medicine,South China Agricultural University,Guangzhou 510642,China)

机构地区：[1]暨南大学基础医学与公共卫生学院,广州510632 [2]华南农业大学兽医学院,广州510642

出　　处：《中华实验眼科杂志》2024年第5期473-477,共5页Chinese Journal Of Experimental Ophthalmology

基　　金：国家自然科学基金(32370878、32061160469)。

摘　　要：基于多能干细胞定向诱导分化视网膜类器官(RO)技术可以高度模拟人类视网膜的发育过程,帮助深入理解视网膜的发育机制,并为视网膜疾病提供新的治疗方法。目前,RO广泛应用于视网膜疾病的机制和治疗研究,尤其在视网膜色素变性(RP)中取得了较为突出的进展。本文总结了利用人多功能干细胞制备RO的方法,阐述了RO-RP疾病模型在PRPF 31、RPGR、CRB 1、RP 2、IMPG 2、NR 2 E 3、USH 2 A、PDE 6 B和TRNT 1等不同突变基因中的机制与治疗应用,概括其在药物筛选、药物毒性试验、基因疗法和细胞疗法方面的研究进展,讨论了RO的研究及应用挑战。After more than ten years of development,retinal organoid(RO)based on pluripotent stem cells can highly simulate the development process of human retina,provide insight into the mechanism of retinal development and provide new treatments for retinal diseases.At present,RO has been widely used in the research of the mechanism and treatment of retinal diseases,especially in retinitis pigmentosa(RP).This review summarizes the methods of preparing RO from human pluripotent stem cells,and elaborates the mechanism and therapeutic application of RO-RP disease model in different mutated genes such as PRPF 31,RPGR,CRB 1,RP 2,IMPG 2,NR 2 E 3,USH 2 A,PDE 6 B and TRNT 1,as well as the research progress of RO in drug screening,drug toxicity testing,gene therapy and cell therapy,and discusses the research and application challenges of RO.

关 键 词：视网膜类器官 视网膜色素变性 疾病模型 

分 类 号：R774.1[医药卫生—眼科]