Defne Amado, MD, PhD

University of Pennsylvania

Dr. Defne Amado is a physician-scientist and Assistant Professor of Neurology at the University of Pennsylvania. Clinically, Dr. Amado cares for people living with amyotrophic lateral sclerosis (ALS). Her translational research lab specializes in the development of novel therapeutics for ALS, as well as for frontotemporal dementia and spinocerebellar ataxia. Her team is particularly interested in using advanced gene editing and gene modulation tools, coupled with novel CNS-targeting delivery methods including evolved AAV capsids, to uncover and treat novel therapeutic targets in neurodegenerative disease pathogenesis using mouse and cell models of disease.