Dr. Chow & Post-doc Bridget Ryan win Research to Watch Award

Dr. Chow and post-doctoral fellow Bridget Ryan are this year's winners of Fighting Blindness Canada's $50,000 Research to Watch Award!

Project: Towards understanding and treating Stargardt disease

Stargardt disease (STGD1) is an autosomal recessive disease with primarily juvenile onset that affects approximately 1:10,000 people and results from mutations in the ABCA4 gene. Although supportive care including the use of sunglasses and reduced vitamin A diets can help slow progression, there are no approved therapies, and all patients are expected to reach legal blindness. Therefore, STGD1 represents an urgent unmet medical need.

The short-term aim of the proposal is to gain insight into the mechanism that drives Stargardt disease pathology. We are utilizing a genetic approach to determine the role of complement system activation (specifically complement factor D which is involved in alternative pathway activation and amplification loop) in Stargardt disease. This research will provide key proof-of-principle data that will guide our long-term aim of developing a gene therapy approach to block the progression of Stargardt disease.

Finally, development of a therapeutic approach to treat Stargardt disease also has the potential for treating those with dry age-related macular degeneration. Although more complex in its etiology, dry AMD and Stargardt disease share key features, namely, a well-established link to the complement system, inflammation and RPE.

Congratulations Bob & Bridget!