As Principal Investigator at UMDNJ-Robert Wood Johnson Medical School for Gene Therapy for Canavan Disease, she leads all research activities at UMDNJ and the affiliated centers. Her research interests have included in vivo neurochemistry of epilepsy and gene transfer approaches for the treatment of neurological disorders. She has published extensively in the field of CNS gene therapy and conducted the first direct gene therapy trial using a liposome-based vector for a pediatric neurodegenerative disorder. In addition to applications of viral vectors and stem cells, Dr. Leone is studying pharmacological approaches in humans and in animal models of Canavan Disease, Amyotrophic Lateral Sclerosis, Tay Sach’s and other neurological disorders.