基因治疗递送系统的研究新进展:遗传性视网膜疾病治疗的曙光  

Advances in Gene Therapy Delivery Systems:Promising Prospects for the Treatment of Inherited Retinal Diseases

作　　者：翟杨 苏子璇 王兴华 姜发纲[2] Zhai Yang;Su Zixuan;Wang Xinghua(The First Clinical College,Tongji Medical College,Huazhong University of Science and Technology,Wuhan 430030,China;Department of Ophthalmology,Union Hospital,Tongji Medical College,Huazhong University of Science and Technology,Wuhan 430022,China)

机构地区：[1]华中科技大学同济医学院第一临床学院,武汉430030 [2]华中科技大学同济医学院附属协和医院眼科,武汉430022

出　　处：《华中科技大学学报（医学版）》2024年第3期414-419,共6页Acta Medicinae Universitatis Scientiae et Technologiae Huazhong

基　　金：国家自然科学基金青年基金资助项目(No.81900912)。

摘　　要：遗传性视网膜疾病是多种先天性视网膜神经退行性疾病的总称,临床上以夜盲、进行性视野缩小、视力下降甚至失明为特点,具有多种遗传形式。由于其病变的根源在于基因突变,通过基因治疗,即利用外源性核苷酸替换或沉默基因缺陷细胞内的致病基因,使细胞表达正确的蛋白质,恢复细胞的功能,就有可能治愈疾病。同时,眼睛具有免疫“豁免”特性,是实现基因治疗的理想器官。为了完成遗传物质的修正,治疗性核苷酸需要进入细胞内发挥作用,携带健康基因的载体递送系统是实现这一过程的有利工具。该文重点总结了包括病毒载体和非病毒载体在内的遗传性视网膜疾病基因治疗递送系统的研究进展及面临的问题。Inherited retinal disease encompasses a range of congenital retinal neurodegenerative conditions,characterized by symptoms such as night blindness,progressive visual field defect,vision loss,and even blindness.The main cause of the disease is gene mutation.Through gene therapy,that is the application of exogenous nucleotides to replace or silence the disease-causing genes in the genetically defective cells,so that the cells can express the correct protein and restore the function of cells.Therefore,it is possible to cure the disease.The immune-privileged status of the eye makes it an ideal organ for gene thera-py.However,successful gene therapy requires delivery systems that can carry therapeutic nucleotides into the cells.This review focuses on the progress and challenges of gene therapy delivery systems for inherited retinal diseases,including viral and non-vi-ral vectors.

关 键 词：遗传性视网膜疾病 视网膜基因治疗 基因递送系统 病毒载体 非病毒载体 

分 类 号：R774.1[医药卫生—眼科]