(S)-PHA533533 Turns On the Paternal Gene Copy of UBE3A (IMAGE)

University of North Carolina Health Care

(S)-PHA533533 Turns On the Paternal Gene Copy of UBE3A

Caption

 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.

Credit

Vihma et. al 2024

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License

CC BY-NC-ND

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