Molecular Therapies for Alport Syndrome and Chronic Kidney Disease

Alport Syndrome (AS) is a genetic disease caused by mutations in genes that form the structural foundations of the kidney, ear, and eye causing them to gradually lose function. The most severe form of the disease affects boys who typically begin to lose their hearing before their teenage years, and as adults, they experience progressive kidney failure. Beyond AS, about 30% of people with chronic kidney disease (CKD) also carry mutations in these genes, making this a broader health challenge. This project will advance new gene  therapies for AS, CKD and other related conditions toward clinical use, with the goal of bringing new treatment options to patients in Minnesota and around the world.