Our mission is to understand disease pathogenesis and to develop next generation genetic medicines for rare inherited disorders, with a particular focus on neurodevelopmental and neurodegenerative conditions. We are working towards creating therapeutics capable of correcting the underlying cause of a disease, targeting its roots at the DNA level.

We use a multitude of complementary genetic, biochemical, molecular and genome engineering approaches to generate and characterize cellular and animal models of disease. Through this, we are gaining insight into how certain mutations lead to dysregulated cellular homeostasis precipitating disease onset. We concentrate our efforts on Niemann-Pick Disease Type C, Duchenne Muscular Dystrophy and Tay-Sachs Disease.