Hôpital Ophtalmique Jules-Gonin, Fondation Asile des Aveugles, Lausanne, Switzerland.
Department of Biomedical and Biotechnological Sciences, School of Medicine, University of Catania, Catania, Italy.
Curr Opin Pharmacol. 2024 Oct;78:102484. doi: 10.1016/j.coph.2024.102484. Epub 2024 Sep 6.
This mini review spotlights the most promising treatments for geographic atrophy, the advanced form of age-related macular degeneration, often resulting in severe and irreversible vision loss. The pathophysiology is complex, and various therapeutic strategies, including anticomplement therapies, gene therapies, cell-based interventions, and artificial intelligence-driven diagnostics are discussed. Anticomplement therapies (antifactors C3 and C5) showed promise in reducing the inflammatory response and the progression of the atrophy. Gene therapies, targeting specific genetic mutations, are under development to correct underlying defects and potentially reverse disease progression. Cell-based therapies are gaining momentum, with early studies indicating encouraging results in the replacement of damaged retinal pigment epithelium cells.
本篇迷你综述聚焦于治疗年龄相关性黄斑变性的晚期形式——地图状萎缩的最有前景的疗法，这种疾病常常导致严重且不可逆转的视力丧失。其病理生理学较为复杂，本文讨论了多种治疗策略，包括抗补体疗法、基因疗法、基于细胞的干预措施和人工智能驱动的诊断。抗补体疗法（抗 C3 和 C5 因子）显示出减轻炎症反应和萎缩进展的潜力。靶向特定基因突变的基因疗法正在开发中，以纠正潜在缺陷并可能逆转疾病进展。基于细胞的疗法也在不断发展，早期研究表明，用受损的视网膜色素上皮细胞替代具有令人鼓舞的结果。
