Zoghbi, Bird Honored for Work on Epigenetics in Neurological Diseases

HOUSTON, Sept. 2, 2026 /PRNewswire/ -- Dr. Huda Zoghbi, founding director of the Duncan Neurological Research Institute (Duncan NRI) at Texas Children's and Distinguished Service Professor and Howard Hughes Medical Institute Investigator at Baylor College of Medicine, along with her long-time collaborator Dr. Adrian Bird, professor at the University of Edinburgh, will be awarded the prestigious 2026 Louisa Gross Horwitz Prize from Columbia University for their pioneering work on epigenetic regulation in the nervous system and its role in human neurological disease, particularly Rett syndrome.

Primarily affecting girls, Rett syndrome is a severe neurodevelopmental disorder that impacts nearly every aspect of life, including the ability to speak, walk, control hand movements, eat, breathe, think, and control seizures. Before the discoveries of Zoghbi and Bird, the disorder was poorly understood and considered untreatable. Today, with lifelong support, individuals with Rett syndrome can live well into adulthood.

Zoghbi discovered in 1999 that mutations in MeCP2 cause Rett syndrome, establishing the first direct link between epigenetics and a severe neurological disorder and enabling early detection of the disease by genetic testing. She also showed that MeCP2 levels must be precisely maintained for neurons to function normally. Too little MeCP2 causes Rett syndrome, while doubling MeCP2 levels causes neurological deficits. Later work revealed that MeCP2 mutations also contribute to a range of other neurological conditions, from autism to juvenile-onset schizophrenia.

Bird uncovered key principles of epigenetic regulation, a chemical process that turns genes on and off without altering their underlying code. In the 1980s, he discovered CpG islands, specialized DNA clusters present near the start of genes. A decade later, his lab identified MeCP2, a protein that scans for chemical tags called methyl groups attached to these clusters and, upon finding them, switches the nearby gene off, acting like a molecular "off switch" for gene activity in neurons and throughout the body.

Though Rett syndrome was thought to cause permanent brain damage, Zoghbi and Bird showed that it could be reversible. Bird's group engineered the first mouse model of Rett syndrome and, in 2007, showed that reactivating MeCP2 in these mice dramatically improved neurological symptoms. Zoghbi's team showed that the effects of abnormal levels of functional MeCP2 can be reversed in mice by oligonucleotide therapy. Based on this work, a new generation of gene replacement (TSHA-102 and NGN-401) and RNA-regulating (ION 440) drugs, are now advancing through clinical development.

"Together, these two scientists have forged a remarkable path from fundamental discovery to therapeutic possibility, exemplifying the highest ideals of biomedical research," said Laura Landweber, chair of Columbia's Horwitz Prize committee.

"Their collective work has transformed our understanding of the molecular basis for a significant human disease and brought renewed hope to patients and families affected by devastating neurological disorders," added Henry Colecraft, a member of the prize committee.

Zoghbi and Bird are the 122nd and 123rd winners of the Horwitz Prize, which is awarded annually by Columbia University for groundbreaking work in medical science. Of the 121 previous Horwitz Prize winners, 55 have gone on to also receive Nobel Prizes.

About Texas Children's
Texas Children's, a nonprofit health care organization, is committed to creating a healthier future for children and women throughout the global community by leading in patient care, education and research. Consistently ranked as the best children's hospital in Texas and among the top in the nation, Texas Children's has garnered widespread recognition for its expertise and breakthroughs in pediatric and women's health. The system includes the Texas Children's Duncan NRI; the Feigin Tower for pediatric research; Texas Children's Pavilion for Women, a comprehensive obstetrics/gynecology facility focusing on high-risk births; Texas Children's Hospital West Campus, a community hospital in suburban West Houston; Texas Children's Hospital The Woodlands, the first hospital devoted to children's care for communities north of Houston and Texas Children's Hospital North Austin, the new state-of-the-art facility providing world-class pediatric and maternal care to Austin families. The organization also created Texas Children's Health Plan, the nation's first HMO focused on children; Texas Children's Pediatrics, the largest pediatric primary care network in the country; Texas Children's Urgent Care clinics that specialize in after-hours care tailored specifically for children; and a global health program that is channeling care to children and women all over the world. Texas Children's Hospital is affiliated with Baylor College of Medicine. For more information, visit www.texaschildrens.org.

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SOURCE Texas Children's Hospital