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Zoghbi named to receive Vanderbilt Prize in Biomedical Science

Dec. 9, 2015, 10:10 AM

Huda Zoghbi, M.D., a physician-scientist known internationally for her extraordinary range of discoveries in neurology and neuroscience, is the recipient of the 2015 Vanderbilt Prize in Biomedical Science, Vanderbilt University officials announced today.

Huda Zoghbi, M.D.

Zoghbi is a professor in the Departments of Pediatrics, Molecular and Human Genetics, Neurology and Neuroscience at Baylor College of Medicine, a Howard Hughes Medical Institute investigator and director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital in Houston.

She is the 10th recipient of the Vanderbilt Prize in Biomedical Science. Established by Vanderbilt University School of Medicine in 2006, the prize honors women scientists with a “stellar record” of research accomplishments who have made significant contributions to mentoring other women in science.

Prize winners receive an honorarium, meet with Vanderbilt faculty, deliver a Flexner Discovery Lecture and mentor Vanderbilt Prize Scholars, women who are pursuing graduate studies in the biomedical sciences in the School of Medicine.

“Huda Zoghbi’s progression from a young woman interested in science to one of the world’s foremost physician-scientists working on the genetic bases of neurological diseases is fascinating and inspiring,” said Lawrence Marnett, Ph.D., associate vice chancellor for Research and senior associate dean for Biomedical Sciences.

“She has a remarkable record in research and training and we look forward to hosting her as she receives the Vanderbilt Prize and begins mentoring the 2015 Vanderbilt Prize Scholar awardee,” Marnett said.

Zoghbi will receive the prize on April 21, 2016, when she is scheduled to give a Flexner Discovery Lecture.

A native of Lebanon, Zoghbi earned a bachelor’s degree in biology with distinction at American University in Beirut. She also began medical school there but because of civil war in Lebanon, transferred to Meharry Medical College in Nashville, where she earned her medical degree in 1979.

As a fellow in Pediatric Neurology at Baylor in October 1983, Zoghbi encountered a 5-year-old girl who had mysteriously lost the ability to speak, walk and use her hands. That week, a paper in the Annals of Neurology described a new entity, Rett syndrome, with the same characteristics.

Her research career was launched.

Sixteen years later, Zoghbi and her colleagues at Baylor reported that Rett syndrome is caused by mutations in an X-linked gene encoding a protein that regulates other gene expression. The discovery opened up a new field of research into disorders of epigenetic regulation.

During this period, she also began investigating spinocerebellar ataxia type 1 (SCA1), a crippling neurodegenerative disease that affects balance and coordination.

After joining the Baylor faculty in 1988, and in collaboration with Harry Orr, Ph.D., at the University of Minnesota, she helped identify the mutant gene responsible for the disorder. Their work shed light on the role that abnormal proteins and protein misfolding play in neurodegenerative diseases like Parkinson’s and Alzheimer’s.

A third line of inquiry led, in the late 1990s, to the discovery that a gene called Math1 is essential for the generation of inner ear hair cells.  The discovery encouraged development of potential gene therapy approaches for deafness.

Zoghbi has received a host of honors for her research including election to the Institute of Medicine and the National Academy of Sciences.

She also is committed to mentoring the next generation of scientists. A quarter of her former trainees are women, several of whom have gone on to leadership positions in academia and industry.

For a complete list of Vanderbilt Prize winners, go to and click on “Vanderbilt Prize.”

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