UBE3A antisense transcript RNA (UBE3A-ATS)

UBE3A antisense transcript RNA (UBE3A-ATS) is a long non-coding RNA expressed primarily from the paternal allele in neurons. It is transcribed antisense across the UBE3A gene locus and acts as an epigenetic silencer by interfering with the transcription of the paternal UBE3A gene, rendering it inactive in neurons. UBE3A protein loss due to this imprinting mechanism, when combined with maternal UBE3A mutations or deletions, causes Angelman syndrome—a severe neurodevelopmental disorder. Unsilencing the paternal UBE3A allele by inhibiting UBE3A-ATS, for example, with antisense oligonucleotides, is a promising therapeutic strategy under active investigation for Angelman syndrome. UBE3A-ATS is therefore considered a clinically actionable target for molecular therapies aiming to restore UBE3A expression and ameliorate disease symptoms[1][4][5][6][7].

Other names

UBE3A-ATSUbe3a-ATS (mouse)Ubiquitin ligase E3A-ATSUBE3A antisense RNALong non-coding UBE3A antisense transcript

Antisense oligonucleotides: Bind to UBE3A-ATS RNA, leading to its degradation or blocking its transcription, thereby *unsilencing* the paternal UBE3A allele and restoring UBE3A protein expression in neurons CRISPR-based genome or transcriptome editing: Target and reduce UBE3A-ATS RNA to relieve suppression of paternal UBE3A ATF/ZF-based transcriptional repression: Target UBE3A-ATS promoter region to inhibit antisense expression