Kelsey J Moody, Jay Tinklepaugh, Elizabeth Obert, Kris Grohn, Jennifer R DeRosa, Ellie Lumen, Brandon Scott Moyer, Scott Campbell, Aaron J Wolfe, Meegan B Sleeper, Anthony H Bianchi, Cheyanne Fisher, Justin Applegate, Emily Leary, Nick LeClair, Danique Wortel, Robert Patrick Doyle, Baerbel Rohrer, Adam R Blanden
Macular degeneration is hallmarked by retinal accumulation of toxic retinoid species (e.g. A2E) for which there is no endogenous mechanism to eliminate. This ultimately results in progressive dysfunction and loss of vision either in advanced age for genetically normal patients (age-related macular degeneration), or in adolescence for those with inherited genetic mutations (Stargardt's disease). Here, we present a proof-of-concept study for an enzyme-based therapy to remove these retinoids, modeled on traditional enzyme replacement therapy...
December 5, 2018: Rejuvenation Research