Article

Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome

2022-06-19

Abstract excerpt

UBE3A encodes ubiquitin protein ligase E3A, and in neurons its expression from the paternal allele is repressed by the UBE3A antisense transcript ( UBE3A-ATS ). This leaves neurons susceptible to loss-of-function of maternal UBE3A . Indeed, Angelman syndrome, a severe neurodevelopmental disorder, is caused by maternal UBE3A deficiency. A promising therapeutic approach to treating Angelman syndrome is to reacti...

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Literature Corpus work: cb2d97c5-2c41-5c4e-b28c-0969bf7f62a3
DOI: 10.1101/2022.06.18.496687

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