Inherited Retinal Degenerations and Non-Neovascular Age-Related Macular Degeneration: Progress and Unmet Needs.

IF 2.6 3区 医学 Q2 OPHTHALMOLOGY Translational Vision Science & Technology Pub Date : 2024-12-02 DOI:10.1167/tvst.13.12.28
Jacque L Duncan, Angela Bowman, Amy Laster, Claire Gelfman, David G Birch, Shannon E Boye, Stephen P Daiger, Lucian Del Priore, Donald J Zack, James T Handa
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Abstract

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.

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遗传性视网膜变性和非新生血管性年龄相关性黄斑变性:进展和未满足的需求。
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。尽管该领域在过去5年中取得了重大进展,但仍存在重大挑战。本文回顾了促进IRD和AMD患者治疗发展的成就和研究进展,包括首个批准的用于rpe65相关视网膜变性的基因增强治疗和用于减缓AMD地理萎缩(GA)进展的补体抑制治疗。这篇文章概述了解决差距和未满足需求的机会,这将导致未来对IRDs和非新生血管性AMD患者治疗的进一步进展。
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来源期刊
Translational Vision Science & Technology
Translational Vision Science & Technology Engineering-Biomedical Engineering
CiteScore
5.70
自引率
3.30%
发文量
346
审稿时长
25 weeks
期刊介绍: Translational Vision Science & Technology (TVST), an official journal of the Association for Research in Vision and Ophthalmology (ARVO), an international organization whose purpose is to advance research worldwide into understanding the visual system and preventing, treating and curing its disorders, is an online, open access, peer-reviewed journal emphasizing multidisciplinary research that bridges the gap between basic research and clinical care. A highly qualified and diverse group of Associate Editors and Editorial Board Members is led by Editor-in-Chief Marco Zarbin, MD, PhD, FARVO. The journal covers a broad spectrum of work, including but not limited to: Applications of stem cell technology for regenerative medicine, Development of new animal models of human diseases, Tissue bioengineering, Chemical engineering to improve virus-based gene delivery, Nanotechnology for drug delivery, Design and synthesis of artificial extracellular matrices, Development of a true microsurgical operating environment, Refining data analysis algorithms to improve in vivo imaging technology, Results of Phase 1 clinical trials, Reverse translational ("bedside to bench") research. TVST seeks manuscripts from scientists and clinicians with diverse backgrounds ranging from basic chemistry to ophthalmic surgery that will advance or change the way we understand and/or treat vision-threatening diseases. TVST encourages the use of color, multimedia, hyperlinks, program code and other digital enhancements.
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