Targeting Lysosomal Dysfunction and Oxidative Stress in Age-Related Macular Degeneration

Age-related macular degeneration (AMD) is the leading cause of vision loss in the Western world, and it currently lacks effective therapy. It is believed that AMD initiates in the aged retinal pigment epithelium (RPE), which presents lysosomal dysfunction and oxidative stress (OxS) that ultimately l...

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Main Authors: Ana S. Falcão, Margarida L. Pedro, Sandra Tenreiro, Miguel C. Seabra
Format: Article
Language:English
Published: MDPI AG 2025-05-01
Series:Antioxidants
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Online Access:https://www.mdpi.com/2076-3921/14/5/596
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author Ana S. Falcão
Margarida L. Pedro
Sandra Tenreiro
Miguel C. Seabra
author_facet Ana S. Falcão
Margarida L. Pedro
Sandra Tenreiro
Miguel C. Seabra
author_sort Ana S. Falcão
collection DOAJ
description Age-related macular degeneration (AMD) is the leading cause of vision loss in the Western world, and it currently lacks effective therapy. It is believed that AMD initiates in the aged retinal pigment epithelium (RPE), which presents lysosomal dysfunction and oxidative stress (OxS) that ultimately leads to RPE damage and AMD progression. AMD is a complex pathology, so multitarget treatments are required to act on different pathways, presenting several challenges. In this review, we discuss the current knowledge on the pathogenesis of this disease, focusing mainly on lysosomal dysfunction and OxS. Because transcription factors regulate homeostasis, the transcription factor EB (TFEB), which controls lysosomal function and biogenesis, and the nuclear factor erythroid 2-related factor 2 (NRF2), which manages OxS, have been proposed as promising targets for disease intervention. Finally, we discuss the interplay of these pathways for a potential synergistic effect on AMD-targeted therapies, as they could change the course of today’s available treatments for AMD.
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series Antioxidants
spelling doaj-art-2ac07d192bb24ad39e7da40d94c4a4f22025-08-20T01:56:17ZengMDPI AGAntioxidants2076-39212025-05-0114559610.3390/antiox14050596Targeting Lysosomal Dysfunction and Oxidative Stress in Age-Related Macular DegenerationAna S. Falcão0Margarida L. Pedro1Sandra Tenreiro2Miguel C. Seabra3iNOVA4Health, NOVA Medical School (NMS), Faculdade de Ciências Médicas (FCM), Universidade Nova de Lisboa, 1169-056 Lisboa, PortugaliNOVA4Health, NOVA Medical School (NMS), Faculdade de Ciências Médicas (FCM), Universidade Nova de Lisboa, 1169-056 Lisboa, PortugaliNOVA4Health, NOVA Medical School (NMS), Faculdade de Ciências Médicas (FCM), Universidade Nova de Lisboa, 1169-056 Lisboa, PortugaliNOVA4Health, NOVA Medical School (NMS), Faculdade de Ciências Médicas (FCM), Universidade Nova de Lisboa, 1169-056 Lisboa, PortugalAge-related macular degeneration (AMD) is the leading cause of vision loss in the Western world, and it currently lacks effective therapy. It is believed that AMD initiates in the aged retinal pigment epithelium (RPE), which presents lysosomal dysfunction and oxidative stress (OxS) that ultimately leads to RPE damage and AMD progression. AMD is a complex pathology, so multitarget treatments are required to act on different pathways, presenting several challenges. In this review, we discuss the current knowledge on the pathogenesis of this disease, focusing mainly on lysosomal dysfunction and OxS. Because transcription factors regulate homeostasis, the transcription factor EB (TFEB), which controls lysosomal function and biogenesis, and the nuclear factor erythroid 2-related factor 2 (NRF2), which manages OxS, have been proposed as promising targets for disease intervention. Finally, we discuss the interplay of these pathways for a potential synergistic effect on AMD-targeted therapies, as they could change the course of today’s available treatments for AMD.https://www.mdpi.com/2076-3921/14/5/596age-related macular degenerationlysosomal dysfunctionNRF2oxidative stressretinal pigment epitheliumTFEB/mTORC1 axis
spellingShingle Ana S. Falcão
Margarida L. Pedro
Sandra Tenreiro
Miguel C. Seabra
Targeting Lysosomal Dysfunction and Oxidative Stress in Age-Related Macular Degeneration
Antioxidants
age-related macular degeneration
lysosomal dysfunction
NRF2
oxidative stress
retinal pigment epithelium
TFEB/mTORC1 axis
title Targeting Lysosomal Dysfunction and Oxidative Stress in Age-Related Macular Degeneration
title_full Targeting Lysosomal Dysfunction and Oxidative Stress in Age-Related Macular Degeneration
title_fullStr Targeting Lysosomal Dysfunction and Oxidative Stress in Age-Related Macular Degeneration
title_full_unstemmed Targeting Lysosomal Dysfunction and Oxidative Stress in Age-Related Macular Degeneration
title_short Targeting Lysosomal Dysfunction and Oxidative Stress in Age-Related Macular Degeneration
title_sort targeting lysosomal dysfunction and oxidative stress in age related macular degeneration
topic age-related macular degeneration
lysosomal dysfunction
NRF2
oxidative stress
retinal pigment epithelium
TFEB/mTORC1 axis
url https://www.mdpi.com/2076-3921/14/5/596
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AT margaridalpedro targetinglysosomaldysfunctionandoxidativestressinagerelatedmaculardegeneration
AT sandratenreiro targetinglysosomaldysfunctionandoxidativestressinagerelatedmaculardegeneration
AT miguelcseabra targetinglysosomaldysfunctionandoxidativestressinagerelatedmaculardegeneration