AGE-RELATED MACULAR DEGENERATION CURRENT INVESTIGATIONS AND TREATMENTS

agerelatedmaculardeg774enerationkapak

GENE THERAPY AND STEM CELL THERAPY IN AGE-RELATED MACULAR DEGENERATION

Fulya Yaylacıoğlu Tuncay

University of Health Sciences, Gülhane Medical Faculty, Department of Medical Biology, Ankara, Türkiye

Yaylacıoğlu Tuncay F. Gene Therapy and Stem Cell Therapy in Age-Related Macular Degeneration. In: Çıtırık M, Şekeryapan Gediz B, editors. AgeRelated Macular Degeneration: Current Investigations and Treatments. 1st ed. Ankara: Türkiye Klinikleri; 2025. p.235-249.

ABSTRACT

Age-related macular degeneration (AMD) is a prevalent chronic degenerative disease of the retina, contributing to severe vision loss in the elderly population. There are two main types of AMD: Non-neovascular (dry) AMD and neovascular (wet) AMD. The conventional treatment options are limited for dry AMD, and the gold standard treatment of wet AMD, anti-VEGFs, creates burdens due to the need for repeated applications. Gene therapy and stem cell therapy provide novel therapeutic options for both dry and wet AMD and have the potential to overcome the limits of conventional treatment methods. Gene therapy was approved for clinical use in a retinal disease in 2017 and gene therapy clinical trials have progressed to phase III for AMD. There is no approved stem cell therapy for retinal diseases; however, several clinical trials explore the safety and efficacy of this approach in AMD. This chapter summarizes the general concepts and clinical trials of gene and stem cell therapies in AMD.

Keywords: Gene therapy; Stem cell therapy; Age-related macular degeneration; Anti-VEGF; Clinical trials

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