Development of stem cell-based gene therapy to treat neuroretinal degeneration

Age-related macular degeneration (AMD) is a progressive retinal disease leading to severe vision loss, with its late-stage forms classified as dry or wet AMD. Current gene and cell therapies have limitations, including immune responses and invasive tissue harvesting. This project aims to develop a novel treatment using urine-derived autologous iPSC-derived retinal pigment epithelial (iRPE) cells, genetically enhanced via non-viral Sleeping Beauty transposon technology to express therapeutic proteins (PEDF and GM-CSF), and to evaluate their protective and regenerative effects in oxidative stress–induced retinal models.