OPDC Seminar - Gene therapy to study and treat Parkinson’s disease

Michael Kaplitt, M.D., Ph.D., is a tenured Professor of Neurological Surgery, Vice-Chairman for Research, and Residency Program Director in the Department of Neurological Surgery at Weill Cornell Medical College. Dr. Kaplitt graduated from Princeton University in 1987 with a degree in Molecular Biology and Russian Studies. Through the Tri-Institutional M.D.-Ph.D. Program, he received his Ph.D. in Molecular Neurobiology in 1993 from The Rockefeller University and his M.D. in 1995 from Cornell Medical College. After his neurosurgery residency at The New York-Presbyterian Hospital, he completed a fellowship in Stereotactic and Functional Neurosurgery at the University of Toronto prior to joining Weill Cornell in July 2001. He has pioneered the use of gene therapy in the brain, having published on the first use of adeno-associated virus (AAV) as a gene delivery vehicle for the brain in 1994. In 2003, he performed the first human clinical trial of gene therapy for Parkinson’s disease, which led to the first successful double-blind, controlled study of gene therapy for a neurological disease. His lab continues to focus upon use of gene transfer to generate new models and novel potential therapies for Parkinson’s disease, psychiatric disorders and pain. He has also pioneered the use of non-invasive focused ultrasound in patients with tremor and Parkinson’s disease, and is currently performing studies in rodents, primates and human subjects exploring novel applications of focused ultrasound in Parkinson’s and Alzheimer’s diseases. Dr. Kaplitt is the recipient of numerous honors and awards, including the Young Investigator Award from the American Society for Gene Therapy, and is a member of the American Society for Clinical Investigation.