Gene therapies aim to deliver a therapeutic payload to specified tissues with underlying protein deficiency. Since the 1990s, gene therapies have been explored as potential treatments for chronic conditions requiring lifetime care and medical management. Ocular gene therapies target a range of ocular disorders, but retinal diseases are of particular importance due to the prevalence of retinal disease and the current treatment burden of such diseases on affected patients, as well as the challenge of properly delivering these therapies to the target tissue. The purpose of this review is to provide an update on the most current data available for five different retinal gene therapies currently undergoing clinical trials for use against age-related macular degeneration (AMD) and the development of novel delivery routes for the administration of such therapies. Research has been performed and compiled from PubMed and the select authors of this manuscript on the treatment and effectiveness of five current retinal gene therapies: Luxturna, ADVM-022, RGX-314, GT-005, and HMR59. We present the available data of current clinical trials for the treatment of neovascular and dry age-related macular degeneration with different AAV-based gene therapies. We also present current research on the progress of developing novel routes of administration for ocular gene therapies. Retinal gene therapies offer the potential for life-changing treatment for chronic conditions like age-related macular degeneration with a single administration. In doing so, gene therapies change the landscape of treatment options for these chronic conditions for both patient and provider.
摘要: 基因治疗的目的是将具备治疗作用的载体运输至有潜在蛋白缺乏的特定组织。自二十世纪九十年代以来, 基因治疗作为有潜力的一种治疗方法而被研究, 用于需要终生治疗和管理的慢性疾病。眼基因治疗可以靶向治疗一系列眼病, 但由于目前视网膜疾病的患病率高、病人治疗的负担大以及将治疗载体送至特定靶组织挑战性大的特点, 基因治疗在视网膜疾病中的作用尤为重要。这篇综述旨在更新与视网膜疾病基因治疗相关的最新数据, 包括正在进行的临床试验、用于年龄相关性黄斑变性 (AMD) 的5种不同治疗方法及基因治疗的新型给药途径等。通过检索PubMed, 本文作者围绕Luxturna、ADVM-022、RGX-314、GT-005和HMR59这五种视网膜基因的治疗方法和治疗有效性进行了研究和汇编。我们提供了目前临床试验的可用数据, 这些试验以不同AAV基因治疗为基础, 用于治疗新生血管和干性年龄相关性黄斑变性。针对眼基因治疗, 我们还介绍了近期新型给药途径的研究进展。视网膜基因治疗为需要终生治疗的慢性疾病 (如年龄相关性黄斑变性) 提供了单次给药的可能性。在此过程中, 基因治疗同时改变了患者和医生对这些慢性疾病的治疗选择。.
© 2021. The Author(s), under exclusive licence to The Royal College of Ophthalmologists.