TERAPIA GÊNICA E TERAPIA CELULAR COM CÉLULAS-TRONCO NA DEGENERAÇÃO MACULAR RELACIONADA À IDADE: AVANÇOS RECENTES E PERSPECTIVAS TRANSLACIONAIS

Isabella Dias Ribeiro; Camilla Izabele Fernandes de Souza; Carolina da Silva Leal; Cristal Wessling Cardoso; Izabela Soares de Oliveira; Mônica Martinez Segura Teixeira Coelho

doi:10.51891/rease.v11i10.21381

Authors

Isabella Dias Ribeiro Universidade de Vassouras
Camilla Izabele Fernandes de Souza Universidade de Vassouras
Carolina da Silva Leal Universidade de Vassouras
Cristal Wessling Cardoso Universidade de Vassouras
Izabela Soares de Oliveira Universidade de Vassouras
Mônica Martinez Segura Teixeira Coelho Universidade de Vassouras

DOI:

https://doi.org/10.51891/rease.v11i10.21381

Keywords:

Age-Related Macular Degeneration. Gene Therapy. Stem Cells.

Abstract

This study aimed to critically review recent evidence on gene and cell therapies for age-related macular degeneration (AMD). An integrative review was conducted in PubMed/MEDLINE, LILACS, SciELO, BVS and ClinicalTrials.gov, between 2020 and 2025, including phase I–III clinical trials, translational studies, and systematic reviews. A total of 25 studies were analyzed. Gene therapy using adeno-associated viral vectors, such as RGX-314, Ixo-vec and 4D-150, showed up to 80% reduction in retreatment need for neovascular AMD, with sustained vision for up to two years. Cell therapy, particularly OpRegen, led to average gains of +6 to +9 letters maintained for 36 months in patients with geographic atrophy, along with OCT-documented structural improvement. Trials with induced pluripotent stem cells confirmed safety, although functional outcomes remain limited. In conclusion, gene and cell therapies represent a milestone in AMD treatment, offering complementary approaches that may constitute the second major revolution in ophthalmology, with clinical, social, and economic impact.