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Baylor neurologist awarded $3 million Breakthrough prize for neuroscience research

Earlier this month, Dr. Huda Zoghbi, pediatric neurologist and pioneering neurogeneticist, was awarded a prestigious Breakthrough prize in Life Sciences at a Silicon Valley ceremony.

Dr. Zoghbi is a professor in the Departments of Pediatrics, Molecular and Human Genetics, Neurology and Neuroscience at Baylor College of Medicine, investigator at the Howard Hughes Medical Institute and director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s where she sees patients. She was awarded for her breakthrough discoveries of the genetic causes and biochemical mechanisms of spinocerebellar ataxia and Rett syndrome. These discoveries have paved the way for understanding other neurodegenerative disorders at large.

Back in October, Baylor published a news release explaining Dr. Zoghbi’s recent research into an approach that could one day help delay or prevent Alzheimer’s disease by taking a pill to decrease accumulation of toxic proteins in the brain. While many researchers in the field focus on the final stages of Alzheimer’s, Dr. Zoghbi and her lab have been focusing on the early stages with the intention of preventing or reducing brain degeneration.

The researchers used enzyme inhibition to determine how to decrease the brain levels of tau, a protein involved in Alzheimer’s Disease and dementia. They found one enzyme, Nuak1, reduced tau levels. The hope is that a drug could one day be made to reduce the levels of tau in the brains of those with dementia risk factors.

The Breakthrough Prizes recognize the world’s top researchers in the areas of life sciences, fundamental physics, and mathematics. Sergey Brin and Anne Wojcicki, Mark Zuckerberg and Priscilla Chan, and Yuri and Julia Milner founded the Breakthrough prizes five years ago.

“Dr. Zoghbi has made seminal discoveries in diverse areas of neuroscience. Her work has significantly furthered the understanding of neurodevelopmental and neurodegenerative disorders and revealed novel strategies to reverse these conditions,” said Dr. Paul Klotman, president, CEO and executive dean of Baylor College of Medicine. “She is a superb scientist, educator and clinician and is most deserving of this award. We’re fortunate that Dr. Zoghbi calls Baylor and Texas Children’s home.”

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