hiPSC-RPE

hiPSC-RPE refers to human induced pluripotent stem cell-derived retinal pigment epithelium cells, a cell-based therapy being developed for advanced retinal degenerative diseases that lead to blindness, such as age-related macular degeneration, Leber congenital amaurosis, retinitis pigmentosa, Stargardt disease, choroideremia, and Vogt-Koyanagi-Harada disease. These cells are designed to replace or support dysfunctional native retinal pigment epithelium (RPE) cells, which are crucial for retinal health and vision. The therapy aims to reconstruct a functional RPE layer, maintain cell polarity, and perform essential RPE functions like phagocytosis of photoreceptor outer segments. Some approaches involve engineered hiPSC-RPE cells, incorporating gene therapy strategies to address specific genetic defects, such as restoring Kir7.1 function in Leber congenital amaurosis type-16. The cells are typically delivered via subretinal transplantation, either as suspensions or as sheets/strips, and are being investigated for their survival, integration, and ability to restore visual function without severe adverse effects.

Other names

human induced pluripotent stem cell-derived retinal pigment epithelium cellshiPSC-derived RPE cellshiPSC-RPE cells