靶向治疗视网膜疾病的研究进展  

Research progress of targeted therapy for retinal diseases

作　　者：潘逸聪 李楚齐 邵毅[1] PAN Yicong;LI Chuqi;SHAO Yi(Department of Ophthalmology,the First Affiliated Hospital of Nanchang University,Nanchang 330006,Jiangxi Province,China)

机构地区：[1]南昌大学第一附属医院眼科,江西省南昌市330006

出　　处：《眼科新进展》2021年第7期692-695,共4页Recent Advances in Ophthalmology

基　　金：中央引导地方科技发展资金(编号20211ZDG02003);江西省重点研发项目(编号20181BBG70004、20203BBG73059)。

摘　　要：作为一种非传统但十分新颖而精准的疗法,基因手段在多种遗传性视网膜疾病的诊治中代表了一种全新而可靠的希望,尤其是先天性黑矇与Stargardt病。近年来,随着临床应用技术和诊断水平的更新及提高,利用基因手段来诊治遗传性视网膜疾病成效颇丰。本文旨在概述测序技术和基因治疗在遗传性视网膜疾病中的作用和最新的研究进展,并对目前使用的主要三种靶向技术进行综述。As a non-traditional but very novel and accurate method,genetic method represents a new and reliable hope in the diagnosis and treatment of most hereditary retinal diseases,especially for Leber’s congenital amaurosis and Stargardt disease.In recent years,with the update and improvement of clinical application technology and diagnosis level,the use of genetic means in the diagnosis and treatment of hereditary retinal diseases has achieved great results.The purpose of this paper is to summarize the role and latest research progress of sequencing and gene therapy in hereditary retinal diseases,and to review the three main targeting techniques used at present.

关 键 词：遗传性视网膜疾病 腺相关病毒 视网膜变性 视网膜下基因治疗 先天性黑矇 

分 类 号：R774[医药卫生—眼科]