Gene therapy for age-related macular degeneration: a promising frontier in vision preservation
Abstract Age-related macular degeneration (AMD) is a leading cause of central vision loss, progressively impairing the retina and affecting millions worldwide. By 2040, global cases of AMD are projected to reach 300 million, posing a significant public health challenge. While early AMD may only caus...
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BMC
2025-05-01
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| Series: | Cell Communication and Signaling |
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| Online Access: | https://doi.org/10.1186/s12964-025-02246-4 |
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| author | Kiavash Hushmandi Hiu Yan Lam Wendy Meihua Wong Wency Tan Seyed-Hashem Daryabari Russel J. Reiter Najma Farahani Alan Prem Kumar |
| author_facet | Kiavash Hushmandi Hiu Yan Lam Wendy Meihua Wong Wency Tan Seyed-Hashem Daryabari Russel J. Reiter Najma Farahani Alan Prem Kumar |
| author_sort | Kiavash Hushmandi |
| collection | DOAJ |
| description | Abstract Age-related macular degeneration (AMD) is a leading cause of central vision loss, progressively impairing the retina and affecting millions worldwide. By 2040, global cases of AMD are projected to reach 300 million, posing a significant public health challenge. While early AMD may only cause mild visual impairment, advanced stages, particularly neovascular (wet) and non-neovascular (dry) AMD, can lead to severe vision loss or legal blindness, substantially affecting daily life. The introduction of anti-angiogenic therapies has revolutionized wet AMD treatment, offering a high probability of preserving or improving vision. However, these therapies do not halt AMD progression, and no definitive treatments exist for dry AMD. The limitations of current therapies, such as frequent injections and treatment resistance, underscore the urgent need for novel strategies. Gene therapy, which has shown success in treating other hereditary retinal diseases, offers a promising long-term solution for AMD by targeting retinal cells to produce therapeutic proteins. This review explores the potential of gene therapy for AMD, examining recent clinical trials and future treatment directions. Graphical Abstract |
| format | Article |
| id | doaj-art-a6ffe7d336bb4db7a709215b100d49a6 |
| institution | Kabale University |
| issn | 1478-811X |
| language | English |
| publishDate | 2025-05-01 |
| publisher | BMC |
| record_format | Article |
| series | Cell Communication and Signaling |
| spelling | doaj-art-a6ffe7d336bb4db7a709215b100d49a62025-08-20T03:48:18ZengBMCCell Communication and Signaling1478-811X2025-05-0123113510.1186/s12964-025-02246-4Gene therapy for age-related macular degeneration: a promising frontier in vision preservationKiavash Hushmandi0Hiu Yan Lam1Wendy Meihua Wong2Wency Tan3Seyed-Hashem Daryabari4Russel J. Reiter5Najma Farahani6Alan Prem Kumar7Nephrology and Urology Research Center, Clinical Sciences Institute, Baqiyatallah University of Medical SciencesDepartment of Pharmacology, Yong Loo Lin School of Medicine, National University of SingaporeCentre for Innovation & Precision Eye Health, Yong Loo Lin School of Medicine, National University of SingaporeDepartment of Pharmacology, Yong Loo Lin School of Medicine, National University of SingaporeBasir Eye Health Research CenterDepartment of Cell Systems and Anatomy, Long School of Medicine, UT Health San AntonioFarhikhtegan Medical Convergence Sciences Research Center, Farhikhtegan Hospital Tehran Medical Sciences, Islamic Azad UniversityDepartment of Pharmacology, Yong Loo Lin School of Medicine, National University of SingaporeAbstract Age-related macular degeneration (AMD) is a leading cause of central vision loss, progressively impairing the retina and affecting millions worldwide. By 2040, global cases of AMD are projected to reach 300 million, posing a significant public health challenge. While early AMD may only cause mild visual impairment, advanced stages, particularly neovascular (wet) and non-neovascular (dry) AMD, can lead to severe vision loss or legal blindness, substantially affecting daily life. The introduction of anti-angiogenic therapies has revolutionized wet AMD treatment, offering a high probability of preserving or improving vision. However, these therapies do not halt AMD progression, and no definitive treatments exist for dry AMD. The limitations of current therapies, such as frequent injections and treatment resistance, underscore the urgent need for novel strategies. Gene therapy, which has shown success in treating other hereditary retinal diseases, offers a promising long-term solution for AMD by targeting retinal cells to produce therapeutic proteins. This review explores the potential of gene therapy for AMD, examining recent clinical trials and future treatment directions. Graphical Abstracthttps://doi.org/10.1186/s12964-025-02246-4Macular degenerationAge-related macular degenerationRetinal diseasesGenetic therapyGene delivery systems |
| spellingShingle | Kiavash Hushmandi Hiu Yan Lam Wendy Meihua Wong Wency Tan Seyed-Hashem Daryabari Russel J. Reiter Najma Farahani Alan Prem Kumar Gene therapy for age-related macular degeneration: a promising frontier in vision preservation Cell Communication and Signaling Macular degeneration Age-related macular degeneration Retinal diseases Genetic therapy Gene delivery systems |
| title | Gene therapy for age-related macular degeneration: a promising frontier in vision preservation |
| title_full | Gene therapy for age-related macular degeneration: a promising frontier in vision preservation |
| title_fullStr | Gene therapy for age-related macular degeneration: a promising frontier in vision preservation |
| title_full_unstemmed | Gene therapy for age-related macular degeneration: a promising frontier in vision preservation |
| title_short | Gene therapy for age-related macular degeneration: a promising frontier in vision preservation |
| title_sort | gene therapy for age related macular degeneration a promising frontier in vision preservation |
| topic | Macular degeneration Age-related macular degeneration Retinal diseases Genetic therapy Gene delivery systems |
| url | https://doi.org/10.1186/s12964-025-02246-4 |
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