遗传性视网膜变性与非新生血管性年龄相关性黄斑变性：进展与未满足的需求
Inherited Retinal Degenerations and Non-Neovascular Age-Related Macular Degeneration: Progress and Unmet Needs.
作者信息
Duncan Jacque L, Bowman Angela, Laster Amy, Gelfman Claire, Birch David G, Boye Shannon E, Daiger Stephen P, Del Priore Lucian, Zack Donald J, Handa James T
机构信息
Wayne and Gladys Valley Center for Vision, Department of Ophthalmology, University of California, San Francisco, San Francisco, CA, USA.
Foundation Fighting Blindness, Columbia, MD, USA.
出版信息
Transl Vis Sci Technol. 2024 Dec 2;13(12):28. doi: 10.1167/tvst.13.12.28.
Inherited retinal degeneration (IRD) disease and age-related macular degeneration (AMD) are leading causes of irreversible vision loss and blindness. Although significant progress has advanced the field in the past 5 years, significant challenges remain. The current article reviews the accomplishments and research advances that have fueled the development of treatments for patients with IRD and AMD, including the first approved gene-augmentation treatment for RPE65-related retinal degeneration and complement inhibition therapies to slow progression of geographic atrophy (GA) in AMD. The article outlines opportunities to address gaps and unmet needs that should lead to additional progress toward the development of treatments for patients with IRDs and non-neovascular AMD in the future.
遗传性视网膜变性（IRD）疾病和年龄相关性黄斑变性（AMD）是导致不可逆视力丧失和失明的主要原因。尽管在过去5年里该领域取得了重大进展，但仍存在重大挑战。本文回顾了推动IRD和AMD患者治疗发展的成就和研究进展，包括首个获批的针对RPE65相关视网膜变性的基因增强治疗以及减缓AMD中地图样萎缩（GA）进展的补体抑制疗法。本文概述了解决差距和未满足需求的机会，这些机会应能在未来推动针对IRD和非新生血管性AMD患者的治疗取得更多进展。
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