Article

Development of an ASO therapy for Angelman syndrome by targeting an evolutionarily conserved region at the start of the <i>UBE3A-AS</i> transcript

2021-07-27

Abstract excerpt

<h4>ABSTRACT</h4> Angelman syndrome is a devastating neurogenetic disorder for which there is currently no effective treatment. It is caused by mutations or epimutations affecting the expression or function of the maternally inherited allele of the ubiquitin-protein ligase E3A ( UBE3A ) gene. The paternal UBE3A allele is imprinted in neurons of the central nervous system (CNS) by the UBE3A antisense ( UBE3A-A...

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Literature Corpus work: 528165e9-f3f4-5031-b974-7f1425dbb73d
DOI: 10.1101/2021.07.27.453820

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