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UBE3A antisense transcript (UBE3A-ATS) is a long non-coding RNA transcribed from the paternal allele at the UBE3A gene locus. It plays a critical role in genomic imprinting and regulation of UBE3A expression, particularly in neurons. Its primary function is the repression of paternal allele expression for UBE3A within neuronal cells. Loss or dysfunction leads to unsilencing/re-expression from this normally silent copy, underlying Angelman syndrome. Therapeutic strategies are being developed to target UBE3A-ATS to reactivate the paternal allele.
Disruption or reduction of UBE3A-ATS activity/expression to reactivate functional protein production from the paternal allele.
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