Huda Zoghbi is Professor of Pediatrics, Molecular and Human Genetics, Neurology, and Neuroscience at Baylor College of Medicine, an Investigator with the Howard Hughes Medical Institute, and the founding Director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital. Zoghbi’s interests range from neurodevelopment to neurodegeneration. Her discovery (with Harry Orr) that Spinocerebellar Ataxia type 1 is caused by expansion of a polyglutamine tract and her subsequent studies that such 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. Zoghbi’s work in neurodevelopment led to the discovery of the gene Math1/Atoh1 and to showing that it governs the development of several components of the proprioceptive, balance, hearing, vestibular, and breathing pathways. Zoghbi’s group also discovered that mutations in MECP2 cause the postnatal neurological disorder Rett syndrome and revealed the importance of this gene for various neuropsychiatric features. Zoghbi trained over 90 scientists and physician-scientists and is a member of several professional organizations and boards. She has been elected to the National Academy of Medicine, the National Academy of Sciences, and the American Academy of Arts and Sciences. Among Dr. Zoghbi’s honors are the Pearl Meister Greengard Prize from Rockefeller University, the Shaw Prize in Life Science and Medicine, the Breakthrough Prize in Life Sciences, Canada Gairdner International Prize, and Honorary degrees from Yale University, Harvard University and the University of Massachusetts Medical School, the Victor A. McKusick Leadership Award, American Society of Human Genetics, and most recently the 2020 Brain Prize, Lundbeck Foundation and the Citation Laureate by Web of Science.

Research Interests

Molecular Mechanisms of Neurodevelopment and Neurodegeneration. Huda Zoghbi wants to understand the mechanisms underlying brain development and degeneration. Working primarily in mouse models and humans, Zoghbi and her team study the activities of proteins involved in neuropsychiatric disorders such as Rett syndrome, MECP2 duplication syndrome, and mania. They also collaborate with labs studying Drosophila to study the function of proteins that accumulate in late-onset degenerative diseases like Alzheimer's and inherited movement disorders such as Spinocerebellar ataxia type 1 (SCA1). The team?s goal is to find ways to reduce abnormal accumulation of proteins in degenerative conditions and normalize protein levels in developmental diseases.

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Primary Section

Section 24: Cellular and Molecular Neuroscience

Secondary Section

Section 41: Medical Genetics, Hematology, and Oncology