antisense oligonucleotide targeting Ube3a-ATS

Antisense oligonucleotides (ASOs) targeting the Ube3a antisense transcript (Ube3a-ATS) represent a class of investigational RNA-targeted therapies designed to treat Angelman syndrome (AS). Angelman syndrome is a severe neurodevelopmental disorder caused by the loss of function of the maternally inherited UBE3A gene. In neurons, the paternal copy of the UBE3A gene is epigenetically silenced by a long non-coding RNA transcript known as Ube3a-ATS. By targeting and triggering the degradation of Ube3a-ATS, these ASOs unsilence the paternal UBE3A allele, thereby restoring the expression of functional UBE3A protein in the central nervous system. Prominent candidates in this class include apazunersen (GTX-102) developed by Ultragenyx, obudanersen (ION582) developed by Ionis Pharmaceuticals, and rugonersen (RO7248824) developed by Roche and Oak Hill Bio. These therapies are typically administered intrathecally.

Other names

UBE3A-ATS ASOUBE-3A-ATS ASOUBE 3A-ATS ASOantisense oligonucleotide targeting UBE3A-ATSantisense oligonucleotide targeting UBE3A antisense transcript