Researchers have identified a small molecule that could lead to a safe and effective treatment for the neurodevelopmental condition known as Angelman syndrome.
gene and characterized by poor muscle control, limited speech, epilepsy, and intellectual disabilities. Though there isn't a cure for the condition, new research at the UNC School of Medicine is setting the stage for one.
"This compound we identified has shown to have excellent uptake in the developing brains of animal models," said Philpot, who is a leading expert on Angelman syndrome."We still have a lot of work to do before we could start a clinical trial, but this small molecule provides an excellent starting point for developing a safe and effective treatment for Angelman syndrome.", mark a major milestone in the field, according to Mark Zylka, W.R. Kenan Jr.
"We previously showed that topotecan, a topoisomerase inhibitor, had very poor bioavailability in mouse models," said Vihma."We were able to show that -PHA533533 had better uptake and that the same small molecule could be translated in human-derived neural cells, which is a huge finding. It means it, or a similar compound, has true potential as a treatment for children.
Birth Defects Epigenetics Diseases And Conditions Disorders And Syndromes Autism Infant And Preschool Learning Brain Injury
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