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补体抑制剂在年龄相关性黄斑变性中的应用:一种潜在的治疗选择。

Complement Inhibitors in Age-Related Macular Degeneration: A Potential Therapeutic Option.

机构信息

Department of Ophthalmology, Beijing Friendship Hospital, Capital Medical University, Beijing, China.

Department of Ophthalmology, Beijing Moslem People's Hospital, Beijing, China.

出版信息

J Immunol Res. 2021 Jul 29;2021:9945725. doi: 10.1155/2021/9945725. eCollection 2021.

Abstract

Age-related macular degeneration (AMD) is a multifactorial disease, which can culminate in irreversible vision loss and blindness in elderly. Nowadays, there is a big gap between dry AMD and wet AMD on treatment. Accounting for nearly 90% of AMD, dry AMD still lacks effective treatment. Numerous genetic and molecular researches have confirmed the significant role of the complement system in the pathogenesis of AMD, leading to a deeper exploration of complement inhibitors in the treatment of AMD. To date, at least 14 different complement inhibitors have been or are being explored in AMD in almost 40 clinical trials. While most complement inhibitors fail to treat AMD successfully, two of them are effective in inhibiting the rate of GA progression in phase II clinical trials, and both of them successfully entered phase III trials. Furthermore, recently emerging complement gene therapy and combination therapy also offer new opportunities to treat AMD in the future. In this review, we aim to introduce genetic and molecular associations between the complement system and AMD, provide the updated progress in complement inhibitors in AMD on clinical trials, and discuss the challenges and prospects of complement therapeutic strategies in AMD.

摘要

年龄相关性黄斑变性(AMD)是一种多因素疾病,可导致老年人视力不可逆丧失和失明。如今,干性 AMD 和湿性 AMD 的治疗方法存在很大差距。占 AMD 近 90%的干性 AMD 仍然缺乏有效的治疗方法。大量的遗传和分子研究证实了补体系统在 AMD 发病机制中的重要作用,这导致了对 AMD 治疗中补体抑制剂的深入探索。迄今为止,在近 40 项临床试验中,已有至少 14 种不同的补体抑制剂被或正在被探索用于 AMD 的治疗。虽然大多数补体抑制剂未能成功治疗 AMD,但其中两种在 II 期临床试验中有效抑制 GA 进展的速度,并且都成功进入 III 期临床试验。此外,最近出现的补体基因治疗和联合治疗也为未来治疗 AMD 提供了新的机会。在这篇综述中,我们旨在介绍补体系统与 AMD 之间的遗传和分子关联,提供补体抑制剂在 AMD 临床试验中的最新进展,并讨论 AMD 补体治疗策略的挑战和前景。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/88d8/8346298/f69fed7bd6d9/JIR2021-9945725.001.jpg

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