Huda Zoghbi, MD

Baylor College of Medicine
Dr. Huda Zoghbi is a Distinguished Service Professor at Baylor College of Medicine, an Investigator with the Howard Hughes Medical Institute, and founding Director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital. Dr. Zoghbi graduated from the American University of Beirut (AUB) and received her medical degree from Meharry Medical College. She joined Baylor College of Medicine for her residency and subsequently obtained postdoctoral research training in molecular genetics. Dr. Zoghbi’s expertise ranges from neurodevelopment to neurodegeneration. She and Dr. Harry Orr discovered that Spinocerebellar Ataxia type 1 is caused by expansion of a polyglutamine tract. Her subsequent studies demonstrating that CAG expansion leads to accumulation of the mutant protein in neurons has had profound ramifications since many late-onset neurological disorders involve similar accumulations of disease-driving proteins and inspired her studies in Alzheimer disease. Dr. Zoghbi and collaborators identified regulators of tau levels providing entry points for development of therapeutics to lower tau. Dr. Zoghbi’s pursuit of the genetic basis of Rett syndrome led to her discovery that mutations in MeCP2 cause this postnatal neurological disorder. Her mechanistic studies highlighted the importance of MeCP2 levels for normal brain function. She and her colleagues also identified the gene Math1/Atoh1 that regulates various components of the proprioceptive, respiratory, and auditory network including tiny inner ear hair cells, essential elements of hearing and balance in mammals.