Dr. Huda Zoghbi stands as a preeminent geneticist whose pioneering investigations have revolutionized our understanding of neurological disorders with genetic origins. She currently serves as a Distinguished Service Professor at Baylor College of Medicine where she holds appointments in Pediatrics, Molecular and Human Genetics, Neurology, and Neuroscience. As Director of the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, she leads a world-class center dedicated to uncovering the molecular mechanisms underlying childhood neurological conditions. Born in Lebanon, Dr. Zoghbi completed her undergraduate education at the American University of Beirut before earning her medical degree from Meharry Medical College in 1979. Her career path transformed during her residency at Baylor College of Medicine when she encountered her first Rett syndrome patient in 1983, which ignited her commitment to unraveling the genetic basis of neurological disorders.
Dr. Zoghbi's research has fundamentally reshaped the field of neurogenetics through her meticulous investigations into several devastating neurological conditions. Her work on Rett syndrome, initiated after her clinical encounter in 1983, has been instrumental in revealing the genetic architecture of this disorder that primarily affects young girls. She has made seminal contributions to understanding spinocerebellar ataxia type 1, identifying critical molecular pathways involved in this progressive neurodegenerative disease. Her research extends to CDKL5 Deficiency Disorder through a collaborative initiative funded by the Loulou Foundation, Baylor College of Medicine, and the Jan and Dan Duncan Neurological Research Institute. These groundbreaking discoveries have provided essential diagnostic tools and therapeutic targets that have transformed clinical approaches to previously intractable neurological conditions.
As a Howard Hughes Medical Institute Investigator since 1996, Dr. Zoghbi has established herself as a visionary leader who seamlessly integrates clinical insights with fundamental genetic research. Her transformative contributions have been recognized with the Breakthrough Prize in Life Sciences and the Shaw Prize in Life Science and Medicine, among other prestigious honors. She currently directs the NIH-funded Baylor College of Medicine Intellectual and Developmental Disabilities Research Center, demonstrating sustained leadership in advancing the field through 2026. Beyond her research, Dr. Zoghbi plays a pivotal role in mentoring the next generation of physician-scientists and shaping scientific priorities through her membership in both the National Academy of Sciences and National Academy of Medicine. Her ongoing work continues to focus on deciphering the complex genetic networks underlying neurological disorders with the ultimate goal of developing targeted interventions for affected children and families worldwide.
