Dr. Huda Zoghbi is a pediatric neurologist, a Howard Hughes Medical Institute investigator; a professor in the departments of Pediatrics, Molecular and Human Genetics, and Neurology and Neuroscience at Baylor College of Medicine; and the founding director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital. Her work has focused on elucidating the mechanisms underlying Rett syndrome and spinocerebellar ataxia. In an interview with JCI Editor-at-Large Ushma Neill, Dr. Zoghbi describes her childhood in Beirut, Lebanon. After her medical studies were interrupted by Lebanon’s civil war, Dr. Zoghbi enrolled at Meharry Medical College. She became interested in pediatric neurological disorders during her residency, when she observed many patients with devastating disorders that appeared to have underlying genetic causes. Dr. Zoghbi also describes her discovery of a genetic cause of Rett syndrome, a null mutation in the methyl-CpG binding protein 2 (MeCP2), which researchers are now trying to target therapeutically.