Huda Zoghbi wants to understand the mechanisms underlying brain development and degeneration. Working primarily in mouse models and humans, Zoghbi and her team study the activities of proteins involved in neuropsychiatric disorders such as Rett syndrome, MECP2 duplication syndrome, and mania. They also collaborate with labs studying Drosophila to study the function of proteins that accumulate in late-onset degenerative diseases like Alzheimer’s and inherited movement disorders such as Spinocerebellar ataxia type 1 (SCA1).  The team’s goal is to find ways to reduce abnormal accumulation of proteins in degenerative conditions and normalize protein levels in developmental diseases.

"Encountering girls with Rett Syndrome - that really inspired me to go into science."

 

Originally from Lebanon, Zoghbi is driven by her experiences with patients; she says one of her greatest achievements in life is training students, fellows, and postdoctoral researchers who will continue to do work that impacts many lives. Learn more about Zoghbi and her work.
HHMI Investigator who developed groundbreaking gene-editing technologies among eight honored for transformative advances in the life sciences. HHMI Investigator Huda Zoghbi says she won’t slow down until she finds a treatment for Rett syndrome, a rare neurodevelopmental disorder that impacts children from a young age. And, after decades of discoveries, Zoghbi and her lab have expanded the scope of their work to take aim at Alzheimer’s, too. HHMI is known for our “people, not projects” philosophy. In this newly launched video series, we’re shining the spotlight on several of our scientists to share their stories and offer a behind-the-scenes glimpse at what makes their work – and their labs – unique. Intensive training in young mice staves off symptoms of a rare neurological disorder that afflicts young girls. The discovery may spark new ideas for treatments in humans. HHMI Investigator Huda Zoghbi is one of seven scientists honored with prestigious awards from Canada’s Gairdner Foundation. HHMI investigators Stephen Elledge, Roel Nusse and Huda Zoghbi are among the scientists honored for transformative advances toward understanding living systems and extending human life. Zoghbi shares Shaw Prize for research leading to discovery of genes and proteins involved in Rett syndrome. Identification of a cell signaling pathway that affects the levels of the toxic protein responsible for the neurodegenerative disease SCA1 could lead researchers to new drugs. HHMI announces the names of scientists selected to pilot a new program devoted to supporting transformative, collaborative research.