Huda Zoghbi, MD, is professor in the Departments of Pediatrics, Molecular and Human Genetics, Neurology, and Neuroscience at Baylor College of Medicine. She is also director of the Jan and Dan Duncan Neurological Research Institute and a Howard Hughes Medical Institute Investigator.

Dr. Zoghbi uses molecular and genetic approaches to understand development and the pathogenesis of specific neurodevelopmental and neurodegenerative conditions. She co-discovered the spinocerebellar ataxia type 1 (SCA1) gene with Harry Orr, PhD, and together they unraveled the pathogenesis of this neurodegenerative disorder. Using forward genetic approaches, Dr. Zoghbi and collaborators identified a pathway that can modulate ataxin-1, the protein driving SCA1. Her discoveries in SCA1 revealed some guiding principles relevant to other neurodegenerative disease and inspired her to identify therapeutic entry points for Parkinson’s disease. Dr. Zoghbi discovered the gene that causes Rett syndrome and contributed to understanding of its pathogenesis.

She has received numerous honors, including election to the Institute of Medicine and National Academy of Sciences.