This invention is a novel target for the treatment of both inherited and age-related macular degeneration (MD). UCSF researchers have shown that target inhibition prevents pathological events in the retinal pigment epithelium (RPE), which is the tissue responsible for nourishment and support of light-sensing photoreceptors. Further, the RPE is the primary site of damage for both Stargardt inherited and age-related MD. Currently, there are no approved therapies for dry age-related MD (which is characterized by focal loss of the RPE) or Stargardt disease.
Discovery/concept. Pathway inhibition has been shown to prevent RPE damage while safeguarding RPE integrity in a mouse model of Stargardt inherited MD and in human donors with age-related MD.
To further develop the technology, including novel small molecule screens.
Available under CDA.