Engineering New Biologic Therapies Delivered by Adenoassociated Viral Vectors for treatment of Neurodegenerative Disorders

Date(s) - 11/27/2017
3:00 pm

Todd E. Golde, M.D., Ph.D., Director, Evelyn F. and William L. McKnight Brain Institute, Professor, Department of Neuroscience, College of Medicine, University of Florida

Engineering New Biologic Therapies Delivered by Adenoassociated Viral Vectors for treatment of Neurodegenerative Disorders.

My laboratory has developed a “toolkit” using recombinant adenoassociated viral (rAAV) vectors that enable delivery of a variety of biomolecules to the Central Nervous System. Using combinations of promoters and capsids, we can restrict delivery to each of the major CNS cell types.  Varieties of delivery methods enable facile preclinical studies targeting the brain, spinal cord, peripheral nerves or muscle.  We also use these vectors to develop 1) new models of neurodegenerative disorders and 2) to probe mechanistic pathways regarding new targets. Finally, we are making a major effort to develop multifunctional biomolecules that we believe are needed to treat brain organ failure that is occurring in Neurodegenerative disorders. I will discuss these rAAV vector technologies in the context of the huge unmet need with respect to effective therapies for Neurodegenerative disorders.


Dr. Golde is a Professor of Neuroscience at the University of Florida.  He was appointed Director of the Evelyn F. and William L. McKnight Brain Institute in December 2016, a position in which he oversees, champions, and facilitates the UF neuroscience research programs.  He currently directs the NIH funded 1Florida Alzheimer’s Disease Research Center.  He was previously the founding director of the Center for Translational Research in Neurodegenerative Disease at UF (2010-16), and prior to that served as Chair of Mayo Clinic’s Department of Neuroscience. Dr. Golde received his MD PhD from Case Western Reserve University. He completed a residency in Laboratory Medicine at University of Pennsylvania. After residency, he moved to Mayo Clinic Florida where he rose from Assistant Professor to both Professor of Neuroscience and chair of Mayo Clinic’s internationally recognized Department of Neuroscience. Dr. Golde has published over 230 peer-reviewed manuscripts, which have been cited over 28,000 times. Dr. Golde is well known for his translational research in Alzheimer’s disease (AD) and his work on immunotherapy for neurodegenerative diseases. His scientific honors include the Paul Beeson Faculty, an Alzheimer’s Association Zenith, and MetLife Foundation Awards. In addition to his research and intramural administrative activities, Dr. Golde is an active advocate for AD and neurodegenerative disease research at the state, national, and international levels serving on numerous boards for scientific associations and various scientific foundations.