katch wreck"Finally, we show that (S)-PHA533533 unsilences paternal UBE3A in Angelman syndrome (AS) patient-derived neurons, highlighting its translational potential. Our findings provide a lead for developing a small molecule treatment for AS that could be safe, non-invasively delivered, and capable of brain-wide unsilencing of paternal #UBE3A."
Ube3a unsilencer for the potential treatment of Angelman syndrome - Nature Communications
Angelman syndrome is a neurodevelopmental disorder caused by the deletion of a single gene. Here, researchers discovered a small molecule that could be delivered peripherally to activate a dormant copy of the gene throughout the brain, providing a potential treatment opportunity.
Autism News International
