视网膜疾病基因治疗递送方式的研究现状与进展  

The status and progress of gene therapy delivery techniques for retinal diseases

作　　者：梁莉聪 佘凯芩 陆方[1] Liang Licong;She Kaiqin;Lu Fang(Department of Ophthalmology,West China Hospital of Sichuan University,Chengdu 610000,China)

机构地区：[1]四川大学华西医院眼科,成都610041

出　　处：《中华眼底病杂志》2024年第1期67-75,共9页Chinese Journal of Ocular Fundus Diseases

基　　金：国家自然科学基金(82201212)。

摘　　要：基因治疗是指将遗传物质引入患者细胞,通过增强、抑制、编辑或引入目标基因以产生治疗或预防的效果。基因治疗为遗传性及获得性视网膜疾病的治疗带来了积极的影响和巨大的潜力。在不断优化基因载体的同时,各种递送方式的探索也为视网膜疾病的基因治疗带来了不同的治疗效果。目前主要采用的递送方式为视网膜下注射、玻璃体腔注射和脉络膜上腔注射;考虑到转染效率和递送方式的安全性,新兴的内界膜下注射和无创基因传递正在研究中。基因递送方式的选择对视网膜疾病基因治疗的安全性及有效性至关重要,其不仅与器械、技术发展有关,更重要的是与腺相关病毒的改造、启动子的选择以及目的基因转染的视网膜特定细胞有关。因此,应当综合考虑上述因素,根据最终基因治疗药物和特定转染细胞进行综合考虑,选择最为合适的基因递送方式。Gene therapy is designed to introduce genetic material into the cells of a patient via virus to enhance,inhibit,edit or add a genetic sequence,results in a therapeutic or prophylactic effect.Gene therapy has brought positive influence and great potential for the treatment of retinal diseases including genetic retinal diseases and acquired retinal diseases.In addition to the constant optimization of gene vectors,the exploration of different drug delivery techniques has brought different therapeutic effects for gene therapy of retinal diseases.The main delivery methods include subretinal injection,intravitreal injection,suprachoroidal injection.Considering the transfection efficiency and safety of delivery methods,emerging sub-inner limiting membrane injection and noninvasive gene delivery are under investigation.The selection of gene delivery method is very important for the safety and effectiveness of gene therapy for retinal diseases.It is not only related to the development of equipment and technology,but also related to the modification of adeno-associated virus,the selection of promoter and the specific retinal cells that the target gene wants to be transfected.Therefore,the most appropriate method of gene delivery should be selected according to the final gene therapy agent and the specific transfected cells after taking all these factors into consideration.

关 键 词：视网膜疾病 基因治疗 基因递送方式 视网膜下注射 玻璃体腔注射 脉络膜上腔注射 综述 

分 类 号：R774.1[医药卫生—眼科]