Huda Y. Zoghbi, MD

Huda Y. Zoghbi, MD

Position: Science Partner

Dr. Huda Y. Zoghbi is a professor in the Departments of Pediatrics, Molecular and Human Genetics, Neurology and Neuroscience at Baylor College of Medicine. She is also an investigator at the Howard Hughes Medical Institute and the director of the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital. At HHMI, Dr. Zoghbi and her collaborators have unraveled the genetic underpinnings of a number of devastating neurological disorders, including Rett syndrome and spinocerebellar ataxia type 1 (SCA1). Their discoveries have provided new ways of thinking about more common neurological disorders, including autism, intellectual disability, and Parkinson’s disease, and could lead to better treatments. Dr. Zoghbi and her colleagues have also identified the mutation responsible for SCA1, one of several polyglutamine neurodegenerative disorders that slowly rob their victims of balance and motor control. Her lab team is searching for compounds that enhance the clearance of mutant proteins in several of these diseases, which might slow disease progression or prevent it altogether.

Involvement: TCG III

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