This National Eye Institute (NEI) Vision Research federal grant award (CFDA 93.867) provides $620,149 to The University of Iowa to conduct research on editing AMD risk alleles in human cells. The project aims to apply genome editing technologies, such as base editing and prime editing, to permanently reduce the risk of age-related macular degeneration (AMD), a leading cause of vision loss. The researchers will edit the pathogenic alleles in two major AMD risk loci (10Q26 and 1Q) in human cells, including induced pluripotent stem cell-derived retinal pigment epithelium, retina, and choroid, as well as donor retinal and choroidal explant cultures. This research is expected to shed light on the impact of the ARMS2 A69S gene on HTRA1 expression and the role of CFH variants in complement deposition, while also exploring the efficacy of advanced gene editing for modifying critical risk genes in AMD. The award period runs from April 1, 2025 to March 31, 2030.
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