Ube3a unsilencer for the potential treatment of Angelman syndrome.

Our results showed that treatment with ( S )-PHA533533 and active analogs 2, 3 , (±)- 4 , and ( S , R )- 5 downregulated the Ube3a-ATS similarly to topotecan, albeit the reduction for compound 2 did not reach statistical significance, likely due to an apparent outlier experiment (Fig. 3b ).

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