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Sackler Prize Honoree Dr. Huda Zoghbi Leads Grand Rounds

Dr. B.J. Casey presents Dr. Huda Zoghbi with a plaque for the Mortimer D. Sackler, M.D. Prize for Distinguished Achievement in Developmental Psychobiology Photo Credit: Meredith Wright

While Rett syndrome, a postnatal neurological disorder that affects about one in 10,000 girls, is rare, research on it and how it affects the brain has led to a number of important findings about more common conditions like autism, schizophrenia and bipolar disorder, said Dr. Huda Zoghbi, who was recently awarded the 2014 Mortimer D. Sackler M.D. Prize for Distinguished Achievement in Developmental Psychobiology.

As part of this prize, Dr. Zoghbi led grand rounds on April 22 at Weill Cornell Medical College's Uris Auditorium on this topic, covering how uncommon neurological conditions can offer insights into more common neuropsychiatric diseases.

Dr. Zoghbi, a pioneer in rare brain disorders, is a professor in the Departments of Pediatrics, Molecular and Human Genetics, Neuroscience, and Neurology at Baylor College of Medicine in Houston. During the grand rounds, she largely focused on her Rett syndrome work and the critical protein product that she found implicated in the disease, but also discussed another gene, Shank3, which causes a host of problems when it is duplicated in a patient. Like Rett syndrome, Shank3 overexpression is found in only a small percent of the population, but is implicated in other conditions, like mania in bipolar disorder, that are "found in one percent of the population or more," Dr. Zoghbi said.

A second and equally important topic was also part of her presentation: how understanding a disease mechanism provides researchers with the foundation upon which they can build translational research.

"Translational research, which takes this basic science from the lab into the clinical setting, is so important when built on a strong foundation of fundamental knowledge," said Dr. Zoghbi, who is also an investigator with the Howard Hughes Medical Institute and the director of the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital. "It becomes all about contemplating how to best help the patient, and that's exactly where I want to be."

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