Dr. Paola Leone’s recent work focuses on the development of a novel gene therapy technology selectively developed to target and repair white matter and prevent disease progression in Canavan Disease. This could also benefit other leukodystrophies.
She is a subject of the documentary film, “Little Heroes”, a documentary inspired by the experiences of seven children and their families confronted with a diagnosis of Canavan disease.
Dr. Paola Leone is a Professor of Cell Biology & Neuroscience at the Rowan University School of Osteopathic Medicine in New Jersey. 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.
Publications and Research
Dr. Leone 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 brain disorders. Dr. Leone is currently pursuing a novel gene therapy strategies with therapeutic potential for Canavan Disease. THIS is what you are helping to fund!
Please take some time to learn more about Dr. Leone – and more of the TEAM working to find a cure. The village standing with the Canavan community is thankful for your work and dedication to helping those afflicted with neurological disorders.