Disorders of glycosphingolipid metabolism
We are interested in lysosomal storage diseases; a family of over 50 individually rare metabolic disorders, that typically have a neurodegenerative clinical course. We are investigating underlying pathogenic processes in these disorders and using this…
Therapy and Biomarkers
From our studies of disease mechanisms in lysosomal disorders we are identifying novel clinical interventions strategies for treating these disorders. We use small molecule drugs to target unique steps in the pathogenic cascade and test…
Lysosomal involvement in more common diseases
Lysosomal dysfunction may not be unique to lysosomal storage diseases. We have have found that lysosomal dysfunction plays a role in more common neurodegenerative diseases, infectious diseases and inflammatory conditions. These findings suggest that therapies…
