Article

Antisense oligonucleotides targeting <i>UBE3A-ATS</i> restore expression of <i>UBE3A</i> by relieving transcriptional interference

2021-07-10

Abstract excerpt

Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by loss of function of the maternally inherited UBE3A allele. In neurons, the paternal allele of UBE3A is silenced in cis by the long noncoding RNA, UBE3A-ATS . Unsilencing paternal UBE3A by reducing UBE3A-ATS is a promising therapeutic approach for the treatment of AS. Here we show that targeted cleavage of UBE3A-ATS using antisense oligon...

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Literature Corpus work: 6022920b-2685-5437-8c30-1ac8c8e0719e
DOI: 10.1101/2021.07.09.451826

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