视网膜病变基因治疗中不同注射方式的应用  

Applications of different delivery approaches in retinal gene therapy

作　　者：周玲玲 沈吟 Zhou lingling;Shen Yin(Eye Center,Renmin Hospital of Wuhan University,Wuhan 430060,China;Medical Research Institute,WuhanUniversity,Wuhan 430071,China)

机构地区：[1]武汉大学人民医院眼科中心,430060 [2]武汉大学医学研究院,430071

出　　处：《临床眼科杂志》2023年第6期571-577,共7页Journal of Clinical Ophthalmology

基　　金：政府间国际科技创新合作重点专项(2017YFE0103400)。

摘　　要：基因治疗是目前遗传病的主要治疗手段和研究方向,其中视网膜基因治疗已成为快速发展的新兴领域,超过40个视网膜基因治疗药物已进入临床试验阶段,治疗策略、载体、免疫反应程度等都可能会影响基因治疗的效果,给药方式也成为关键因素。局部给药包括视网膜下注射、玻璃体腔注射和脉络膜上腔注射,是目前视网膜基因治疗主要的三种给药方式,不同注射方式的操作、转导效率和给药后的免疫反应均有所不同,最终达到的治疗效果也存在差异,根据视网膜病变的特点选择合适的给药方式将有助于提高基因治疗效果,本文将对视网膜基因治疗领域三种给药方式的特点、优势、局限性和面临的挑战进行综述。Gene therapy is the main treatment and research orientation of genetic diseases.Retinal gene therapy has become a fast⁃growing emerging field.Over 40 retinal gene therapy drugs have entered the stage of clinical trials.The efficacy of gene therapy might be influenced by various factors,such as therapeutic strategies,vectors and degree of im⁃mune response.The route of administration has also become a key factor for the success of treatment.Topical administra⁃tion includes subretinal injection,intravitreal injection and suprachoroidal injection,which are the three main routes of de⁃livery in retinal gene therapy.The operation,transduction efficiency,and immune response after administration vary ac⁃cording to the delivery approach.There are also differences in the final treatment effects achieved.According to the charac⁃teristics of retinopathy,it’s advisable to select an appropriate route of delivery,which can help improve the efficacy of gene therapy.We will review the characteristics,advantages and challenges of the three delivery approaches in retinal gene ther⁃apy.

关 键 词：视网膜疾病 基因疗法 注射 视网膜下腔 玻璃体腔 脉络膜上腔 

分 类 号：R774.1[医药卫生—眼科]