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Huda Y. Zoghbi, MD, is Professor of Pediatrics, Neurology, Neuroscience, and Molecular and Human Genetics at Baylor College of Medicine, an Investigator with the Howard Hughes Medical Institute, and Director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital.

Zoghbi’s clinical encounters with young girls with Rett syndrome inspired her to go into basic research. Her laboratory ultimately discovered the genetic cause of Rett syndrome and provided insight into the function of the gene in various neurons. Her discovery (with Harry Orr) that Spinocerebellar Ataxia type 1 is caused by expansion of a polyglutamine tract and her studies that such expansion leads to accumulation of the mutant protein has informed studies of other neurodegenerative disorders. Zoghbi also discovered Math1/Atoh1 and showed that it governs the development of several components of the proprioceptive, balance, hearing, vestibular, and breathing pathways.

Dr. Zoghbi was elected to the National Academy of Medicine, the National Academy of Sciences, and the American Academy of Arts and Sciences. Among Zoghbi’s recent honors are the Shaw Prize in Life Science and Medicine, and the Breakthrough Prize in Life Sciences.
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This event was moderated by Anna Greka, MD, PhD, Associate Professor of Medicine at Harvard Medical School and Brigham and Women’s Hospital, and Institute Member of the Broad Institute of MIT and Harvard. She currently sits on the ASCI Council, is Co-Chair of the ASCI Physician-Scientist Development Committee, and is and is a 2018 recipient of the Seldin~Smith Award for Pioneering Research.

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Fetal therapies for single-gene disorders

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