UCL Institute of Ophthalmology, University College London, London, UK.
Moorfields Eye Hospital, London, UK.
Br J Ophthalmol. 2021 Feb;105(2):151-157. doi: 10.1136/bjophthalmol-2020-316195. Epub 2020 Apr 8.
Age-related macular degeneration (AMD) is one of the leading causes of irreversible blindness in the developed world. Antivascular endothelial growth factor therapy has transformed the management and outcome of neovascular AMD (nAMD), although the need for repeated intravitreal injections-even lifelong-and the related complications, high drug costs, frequent clinic visits and repeated imaging have resulted in an enormous burden both to healthcare systems and patients. The application of gene therapy approaches for sustained delivery of a range of antiangiogenic proteins has the promise of helping to address these aforementioned challenges. A number of early phase clinical trials of gene therapy in nAMD have provided encouraging results, with many more ongoing or anticipated. There remain significant areas of controversy, including regarding the optimal treatment targets, routes of administration and potential safety concerns. In this review we aim to provide an update of the current status of gene therapy for nAMD and briefly discuss future prospects.
年龄相关性黄斑变性（AMD）是发达国家导致不可逆性失明的主要原因之一。抗血管内皮生长因子治疗改变了新生血管性 AMD（nAMD）的治疗管理和预后，尽管需要反复进行玻璃体内注射，甚至终生治疗，并且相关的并发症、高昂的药物费用、频繁的就诊和重复的影像学检查给医疗保健系统和患者带来了巨大的负担。基因治疗方法的应用可实现持续输送多种抗血管生成蛋白，有望有助于解决上述挑战。多项 nAMD 基因治疗的早期临床试验提供了令人鼓舞的结果，还有更多的试验正在进行或即将进行。仍存在一些具有争议的重大领域，包括最佳治疗靶点、给药途径和潜在的安全问题。在本文中，我们旨在提供 nAMD 基因治疗的最新现状，并简要讨论未来的前景。
