Boosting a key brain protein could help treat Rett syndrome

 Boosting a key brain protein could help treat Rett syndrome

Researchers have discovered a new way to increase a key brain protein damaged in Rett syndrome, a rare genetic disorder that affects thousands of children worldwide. Early studies in mice and patient-derived cells show the approach can restore normal brain cell function, raising hopes for future therapies.
Scientists at Texas Children’s Duncan Neurological Research Institute and Baylor College of Medicine have uncovered a promising new strategy that could one day help treat Rett syndrome, a rare genetic brain disorder that mainly affects girls and currently has no cure. Credit: AI/ScienceDaily.com


Researchers at Texas Children's Duncan Neurological Research Institute (NRI) and Baylor College of Medicine have reported a promising experimental strategy that could eventually help treat Rett syndrome. Their findings, published in Science Translational Medicine, describe a potential way to increase levels of a key brain protein that is disrupted in the disorder. The work offers early hope for addressing a rare neurodevelopmental disease that currently has no cure.


"Rett syndrome is a rare genetic neurodevelopmental condition that causes a regression in development, typically after 6 to 18 months of normal growth, leading to severe impairments in motor skills, speech and communication," said corresponding author Dr. Huda Zoghbi, director of the Duncan NRI, Distinguished Service Professor at Baylor, and a Howard Hughes Medical Institute investigator. "The disorder primarily affects girls; about 1 in 10,000 live births."

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