Abstract
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.
MeSH terms
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Alleles*
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Animals
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Female
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Gene Silencing / drug effects*
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Male
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Neurons / drug effects*
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Neurons / metabolism*
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Topoisomerase Inhibitors / pharmacology*
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Ubiquitin-Protein Ligases / genetics*
Substances
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Topoisomerase Inhibitors
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Ube3a protein, mouse
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Ubiquitin-Protein Ligases