NIH R01 · 2025
Gene therapy targeting CFH and lipoprotein dynamics in AMD
Gene therapy targeting CFH and lipoprotein dynamics in AMD ABSTRACT Age-related macular degeneration (AMD) is the most common cause of irreversible blindness among the elderly in industrialized countries, and there are no treatments for the majority of patients. Early hallmarks of AMD are the formation of lipid- and protein-rich, extracellular sub-retinal pigmented epithelium (RPE) drusen and basal laminar deposits that contain many constituents that are attributable to the activation of the complement cascade and lipid metabolism. Complement and lipoprotein pathway genes have also been independently associated with AMD through genetic risk association and epidemiological studies. Efforts…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.