Credit: Vihma et al 2024.
(S)-PHA533533 turns on the paternal gene copy of UBE3A. Neurons with a fluorescent reporter for paternal UBE3A gene activity show only a background low fluorescent glow when treated with a vehicle control, but show a bright fluorescent glow when treated with (S)-PHA533533, indicating that the small molecule potently activated the dormant paternal allele of UBE3A.