ALPK1 and retinal degeneration: To test DF-003 in vivo, we established a ROSAH mouse model that was designed to mimic the human genetic disease as heterozygous carriers of one copy of human ALPK1T237M and one copy of wild-type human ALPK1. Kozycki et al. also previously generated knock-in mice bearing the p.T237M mutation in the murine Alpk1 gene3, and while their mice exhibited elevated serum concentrations of chemokines including CCL2, CXCL1, and CXCL10, no changes in spleen size/weight or retinal degeneration were observed through 12 months of age.