Age-related macular degeneration (AMD) is a leading cause of irreversible vision loss in the elderly, characterized by progressive degeneration of the retinal pigment epithelium (RPE) and photoreceptors in the macula. Current treatment options primarily focus on slowing disease progression in neovascular AMD, while effective therapies for dry AMD remain limited. Optogenetics, a revolutionary technique utilizing light-sensitive proteins (opsins) to control the activity of genetically targeted cells, has emerged as a promising therapeutic strategy for restoring vision in retinal degenerative diseases. In retinal disease models, adeno-associated viruses (AAVs) serve as delivery vectors via intravitreal or subretinal injections. This review explores the principles of optogenetics, its application in preclinical AMD models, and the potential for clinical translation of this approach. We discuss the various optogenetic tools, delivery methods, and the challenges and future directions in harnessing this technology to combat AMD-related vision loss.

Optogenetics: A Novel Therapeutic Avenue for Age-Related Macular Degeneration / Grenga, Pier Luigi; Ciancimino, Chiara; Meduri, Alessandro; Fragiotta, Serena. - In: BIOMOLECULES. - ISSN 2218-273X. - 15:9(2025), pp. 1-18. [10.3390/biom15091286]

Optogenetics: A Novel Therapeutic Avenue for Age-Related Macular Degeneration

Grenga, Pier Luigi;Ciancimino, Chiara;Fragiotta, Serena
2025

Abstract

Age-related macular degeneration (AMD) is a leading cause of irreversible vision loss in the elderly, characterized by progressive degeneration of the retinal pigment epithelium (RPE) and photoreceptors in the macula. Current treatment options primarily focus on slowing disease progression in neovascular AMD, while effective therapies for dry AMD remain limited. Optogenetics, a revolutionary technique utilizing light-sensitive proteins (opsins) to control the activity of genetically targeted cells, has emerged as a promising therapeutic strategy for restoring vision in retinal degenerative diseases. In retinal disease models, adeno-associated viruses (AAVs) serve as delivery vectors via intravitreal or subretinal injections. This review explores the principles of optogenetics, its application in preclinical AMD models, and the potential for clinical translation of this approach. We discuss the various optogenetic tools, delivery methods, and the challenges and future directions in harnessing this technology to combat AMD-related vision loss.
2025
adeno-associated viruses (AAVs); age-related macular degeneration; optogenetics; treatment
01 Pubblicazione su rivista::01g Articolo di rassegna (Review)
Optogenetics: A Novel Therapeutic Avenue for Age-Related Macular Degeneration / Grenga, Pier Luigi; Ciancimino, Chiara; Meduri, Alessandro; Fragiotta, Serena. - In: BIOMOLECULES. - ISSN 2218-273X. - 15:9(2025), pp. 1-18. [10.3390/biom15091286]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1748733
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