Storing Fat in the Eye: A Pathway for Tackling AMD

This project aims to manipulate lipid droplet dynamics in a manner that promotes retinal pigment epithelial lipid degradation rather than lipid secretion as a therapeutic strategy for age-related macular degeneration.

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

The mechanism of lipid droplet formation and lipid release in the RPE and its role in RPE homeostasis in AMD has not been well studied. As lipid droplet pathways are ripe with druggable targets, examining RPE lipid droplets offers a rich landscape in which to explore AMD therapeutics.

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.