Results for Lysosomal Storage Diseases

Dr. Davidson works to understand the molecular basis of childhood onset neurodegenerative diseases and the development of gene and small molecule therapies for treatment. She also focuses on how noncoding RNAs participate in neural development and neurodegenerative disease processes, and how they can be harnessed for therapies.

Dr. Ahrens-Nicklas works to understand why patients with inherited biochemical disorders often suffer severe, untreatable neurologic and cardiac symptoms. She strives to elucidate the link between biochemistry and network excitability, in order to drive new approaches to therapy.

Dr. Tricoli's research involves developing a new generation of lentiviral vectors that will prove to limit toxicity and improve activity of the ARSA enzyme, extending the efficacy of and eligibility for gene therapy in metachromatic leukodystrophy, a devastating childhood lysosomal storage disease.