基因治疗在眼底血管性疾病中的应用和展望  

Application and prospect of gene therapy for fundus vascular diseases

作　　者：张朝阳 张敬香[2] 张敬法 Chao-Yang Zhang;Jing-Xiang Zhang;Jing-Fa Zhang(Department of Ophthalmology,Shanghai General Hospital,National Clinical Research Center for Eye Diseases,Shanghai Key Laboratory of Ocular Fundus Diseases,Shanghai Engineering Center for Visual Science and Photomedicine,Shanghai Engineering Center for Precise Diagnosis and Treatment of Eye Diseases,Shanghai 200080,China;Department of Nursing,the People’s Hospital of Huangdao District,Qingdao,266400,Shandong Province,China)

机构地区：[1]中国上海市第一人民医院眼科、国家眼部疾病临床医学研究中心、上海市眼底病重点实验室、上海眼视觉与光医学工程技术研究中心、上海市眼科疾病精准诊疗工程技术研究中心,200080 [2]中国山东省青岛市黄岛区人民医院护理部,266400

出　　处：《国际眼科杂志》2023年第3期400-406,共7页International Eye Science

基　　金：国家自然科学基金面上项目(No.82171062)。

摘　　要：眼底血管性疾病,如新生血管性年龄相关性黄斑变性(nAMD)和糖尿病视网膜病变(DR)是全球范围内患者视力损害的主要病因。随着全球人口老龄化进程和糖尿病发病率的不断提高,这两种眼底疾病的患病率也将持续攀升。当前对眼底血管性疾病的治疗以眼内注射抗血管内皮生长因子(anti-VEGF)药物为主,但存在频繁注射、费用高昂、患者依从性差等问题,因此需要探寻更加持久且有效的治疗策略。基因治疗在遗传性视网膜疾病(IRDs)中的成功应用为眼底血管性疾病的治疗提供了新思路。随着多项临床试验的开展,眼底血管性疾病的基因治疗有望实现临床转化,但仍存在一些问题,包括最佳治疗靶点选择、给药途径和潜在的安全性等。本文重点阐述基因补充和基因编辑技术介导的anti-VEGF治疗在nAMD和DR中的应用和展望。·Fundus vascular diseases,including neovascular age-related macular degeneration(nAMD)and diabetic retinopathy(DR),are the leading causes of visual impairment worldwide.With the accelerated aging and increased incidence of diabetes,the prevalence of these two fundus diseases will continue to rise.Currently,intraocular injection of anti-vascular endothelial growth factor(anti-VEGF)remains the first-line treatment for fundus vascular diseases,but disadvantages exist,such as frequent intraocular injections,high cost and poor compliance,thus more durable and effective therapeutic strategies need to be explored.The successful application of gene therapy in inherited retinal diseases(IRDs)provides a new idea for the treatment of fundus vascular diseases.With the ongoing of several clinical trials,gene therapy for fundus vascular diseases is expected to be employed in the clinical setting.But there still remain some concerns,including the optimal therapeutic targets selection,administration route and safety issues.This review focuses on the application and prospect of gene augmentation and gene editing-mediated anti-VEGF therapy for the treatment of nAMD and DR.

关 键 词：基因治疗 眼底血管性疾病 新生血管性年龄相关性黄斑变性 糖尿病视网膜病变 糖尿病黄斑水肿 抗血管内皮生长因子 

分 类 号：R773.4[医药卫生—眼科] R450[医药卫生—临床医学]