Huda Zoghbi, M.D., is 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. 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 team provided the proof-of-concept data showing that antisense sense oligonucleotide therapy can normalize MeCP2 levels and reverse phenotypes of a MECP2 duplication mouse model.
Dr. Zoghbi has trained over 110 scientists and physician-scientists and is committed to educating the next generation. She has been elected to the National Academy of Medicine, the National Academy of Sciences, and the American Academy of Arts and Sciences. Dr. Zoghbi’s honors include the Breakthrough Prize in Life Sciences; the Canada Gairdner International Prize; the Lundbeck Foundation’s 2020 Brain Prize; the Kavli Prize, and most recently the August M. Watanabe Prize in Translational