Integrated analysis of genetic and clinical data for rational clinical trials of Stargardt disease

Dr. Allikmets and his team will recruit and evaluate patients and family members with ABCA4 disease and associated maculopathies, perform clinical studies to determine quantifiable biomarkers, determine the interplay of ABCA4 variation with that in other genes (i.e., modifiers) resulting in specific sub-phenotypes and sub-categories (slow and fast progressors,) and will search for new genes modifying ABCA4 disease.