Defne Amado, MD, PhD

University of Pennsylvania

Defne Amado is a physician-scientist and Assistant Professor of Neurology at the University of Pennsylvania. She earned her BA in Neuroscience at Columbia University, followed by an MD/PhD in Neuroscience at the University of Pennsylvania. Her thesis work with Dr. Jean Bennett focused in part on the development of Luxturna, the first FDA-approved gene therapy for an inherited disease (Leber’s Congenital Amaurosis). She remained at the University of Pennsylvania for her Neurology residency and Neuromuscular fellowship, turning her focus towards neurodegenerative disease in postdoctoral fellowships with Dr. Alice Chen-Plotkin and Dr. Beverly Davidson. Dr. Amado specializes in the use of gene therapy for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and spinocerebellar ataxia (SCA), both in terms of disease mechanism discovery and therapeutic development. Her lab 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 ALS, FTD, and SCA pathogenesis using mouse and cell models of disease. Clinically, she sees patients with ALS at Penn’s Comprehensive ALS Multidisciplinary Center.


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