Paola Leone, PhD

Canavan Disease Expert

Dr. Leone is a professor of cell biology & neuroscience at the Rowan University School of Osteopathic Medicine (NJ) where she directs all research activities at the cell and gene therapy center. Her research expertise includes in vivo neurochemistry of disease circuits in the brain and gene transfer approaches for the treatment of neurological disorders. She has published extensively in the field of central nervous system (CNS) gene therapy and headed both the first clinical gene therapy trial for a brain disease using a liposome-based vector and the first clinical application of Adeno-Associated Viral (AAV) vectors in the human brain for Canavan disease. Dr. Leone’s efforts in the translational neuroscience sphere have been at the forefront of gene delivery technology development for application to CNS disorders. Dr. Leone is currently pursuing novel pharmacological and gene therapy strategies with therapeutic potential in a wide range of model systems of neurodegenerative disease, including Canavan disease, amyotrophic lateral sclerosis, Alzheimer’s disease, Parkinson’s disease & traumatic brain injury.