Storing Fat in the Eye: A Pathway for Tackling AMD

Project Summary

Age-related macular degeneration (AMD) is characterized by the buildup of fatty deposits outside a cell layer in the back of the eye called the retinal pigment epithelium (RPE). As part of its normal function, the RPE consumes enormous amounts of fat each day, and this fat is temporarily stored inside the cell in spheres called lipid droplets. The researchers’ goal is to understand how the RPE forms lipid droplets and manipulate those lipid droplets in such a way that less fat is secreted from the RPE to form the toxic fatty deposits outside the cell that characterize AMD.

Targeting enzymes governing lipid droplet formation and degradation has become a viable therapeutic strategy in other diseases. RPE lipid droplets may be critical guardians against the onset of AMD. Lipid droplets are highly druggable, with available small molecules that are highly specific to target enzymes required for lipid droplet formation and lipid release, providing an innovative new therapeutic approach for AMD.