Huda Zoghbi, MD

Dr. Huda Zoghbi earned her bachelor of science degree in biology from the American University of Beirut (Lebanon). She enrolled in medical school at the American University of Beirut, but due to the war in Lebanon, Dr. Zoghbi transferred to Meharry Medical College in Nashville, Tennessee, where she earned her medical degree. After residency and postdoctoral research training at Baylor College of Medicine (Houston), she joined the Baylor faculty in 1988. Dr. Zoghbi became an investigator with the Howard Hughes Medical Institute in 1996. In 2010, she became the founding Director of the Jan and Dan Duncan Neurological Research Institute. Dr. Zoghbi has made a series of seminal contributions to our understanding of inherited neurological disorders. Since identifying an expanded trinucleotide repeat as the cause of spinocerebellar ataxia type 1 (SCA1), in collaboration with Dr. Harry Orr, she has led the polyglutamine field in understanding the role of protein misfolding in pathogenesis and provided a molecular mechanism to show how the gain of function in ataxin1 involves enhanced function. More recently, her team and collaborators highlighted the importance of protein levels in this class of disorders and developed a strategy to uncover new druggable targets for SCA1 and other degenerative diseases, such as Alzheimer disease, that are driven by enhanced protein levels or activities.  Dr. Zoghbi also discovered that the sporadic disease Rett syndrome is caused by mutations in MECP2 [methyl CpG binding protein 2] and provided insight into the effect of MECP2 dysfunction on various neurons. Zoghbi’s studies of normal brain development have also, inevitably, led back to the clinic: her work on the Math1 gene has shed light on hearing, proprioception, neonatal breathing, and medulloblastoma.