John J. Gargus, PhD
Dr. Gargus is a highly respected and world renown geneticist who has dedicated his career to advancing scientific research on neurological disorders.
He most recently served as the Director of UC Irvine’s Center for Autism Research and Translation (CART) where he led a group of 60 UCI faculty working together on a platform for drug discovery in neurodevelopmental disorders within the Center for Autism and Neurodevelopmental Disorders. He is PI of two industry-sponsored first-in-human clinical trials for novel molecular therapeutics targeted to genetic diseases of intracellular organelles and was sole USA PI on the pivotal CL-03 Synageva/Alexion clinical trial for Kanuma. He holds 3 patents on novel genetic disease diagnostics and has received major awards from the American Heart Association and the National Headache Foundation for his work.
He received his MD-PhD in Human Genetics from Yale University School of Medicine, carried out a Residency in Pediatrics and a Fellowship in Medical Genetics, also at Yale, and is an ABMG certified medical geneticist. Early in his career he played a leading role introducing molecular genetics into the analysis of physiological processes through symposia he organized and invited reviews. He has received research support for his work from the NIH, Autism Speaks, Doris Duke Foundation and The Thompson Family Foundation.
He joined the University of California, Irvine School of Medicine in 1992 following prior appointments at Emory and Yale. He is Professor of Human Genetics, Pediatrics and Physiology & Biophysics. His clinical subspecialty is inborn errors of metabolism, bioenergetics, and membrane function in the diagnosis of complex neurodevelopmental syndromes. His major research thrust is molecular pathophysiology of the common complex polygenic disease’s migraine and autism, early recognizing the role of channelopathy pathogenesis in common neuropsychiatric diseases. This work provided the foundations for CART for which he led the effort to bring 28 million dollars in philanthropic support.
He has been an invited speaker at several national autism meetings and has been organizer and chair of two large early international society symposia, one of biophysicists and one of geneticists, focusing on cell signaling defects in autism. He additionally serves on the Scientific Advisory Board of the Channelopathy Foundation headquartered in Zurich, Switzerland and several national review groups including prior membership on NIH, ACS, AHA, and Autism Speaks review panels and Chair of several Congressionally Directed Peer Reviewed Medical Research Program panels.