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Angelman syndrome

Drugs to awaken a paternal gene

Mutations in the maternal copy of the UBE3A gene cause a neurodevelopmental disorder known as Angelman syndrome. Drugs that activate the normally silenced paternal copy of this gene may be of therapeutic value. See Letter p.185

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Figure 1: Differential regulation of maternal and paternal UBE3A.

Notes

  1. *This article and the paper1 under discussion were published online on 21 December 2011.

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Correspondence to Arthur L. Beaudet.

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Beaudet, A. Drugs to awaken a paternal gene. Nature 481, 151–152 (2012). https://doi.org/10.1038/nature10784

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