Linda Restifo, MD, PhD

2019 Precision Medicine Symposium

Untangling the Gordian knot of Alzheimer’s disease

Linda Restifo, MD, PhD

Professor, Neurology, Neuroscience, Cellular and Molecular Medicine
Member, Center for Applied Genetics and Genomic Medicine
Member, Evelyn F. McKnight Brain Institute and BIO5 Institute


Dr. Linda Restifo earned all three of her academic degrees from the University of Pennsylvania – BA (Biology), MD, and PhD (Genetics). Her postgraduate clinical training in adult and child neurology was at Harvard Medical Center’s Longwood campus. She did postdoctoral research training in developmental neurogenetics at Brandeis University. Dr. Restifo now directs a research program on developmental brain disorders using the fruit fly (Drosophila melanogaster) genetic model system, with an emphasis on how mutations and environmental toxins disrupt neuronal differentiation. Her goal is to make intellectual disability (a/k/a mental retardation) and autism treatable with safe and effective drugs. With funding from NIH and Autism Speaks, Dr. Restifo and colleagues developed a novel cellular bioassay, based on primary culture of developing brain neurons, which reveals defects caused by mutations or neurotoxins. Their published proof-of-concept drug screen identified potential treatments for developmental brain disorders. Work in progress will determine which genetic defects are most likely to respond to which drugs. Such treatments would represent a dramatic improvement in the medical care of children with cognitive or behavioral delay due to transmitted or de novo mutations. Her collaborators include human geneticists, computer scientists, computational chemists, mechanical engineers, physicians in neurology and psychiatry, as well as other neuroscientists. In the teaching arena, Dr. Restifo is highly motivated to bridge the cultural divide between the worlds of clinical medicine and laboratory science that slows progress in translational medicine.