The role of complement factor D (adipsin) in the pathogenesis of the Stargardt phenotype in Abca4-deficient mice
Stargardt disease affects approximately 1:10,000 individuals and causes progressive, irreversible blindness. While primarily a juvenile disease, age of onset can range from childhood to adulthood. Although supportive care can help slow progression, there are currently no approved therapies and all patients are expected to reach legal blindness. Therefore, Stargardt disease represents an urgent unmet medical need.Oak Bay Biosciences, based in Victoria BC, is a preclinical biotechnology company with a goal to develop the first approved therapy for Stargardt disease. The company’s lead molecule blocks the function of Complement Factor D, a molecule proposed to be the key mediator of retinopathy in Stargardt disease. This project represents a collaboration between Oak Bay Biosciences, Inc. and the laboratory of Dr. Bob Chow at the University of Victoria. In this project, we will use genetic methods to directly test whether removing factor D function in the mouse model of Stargardt disease prevents the progression of blindness.