Oligonucleotides for inducing paternal UBE3A expression

US12596118B2 · 04/07/2026

FIG. 4 · Sheet 4

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The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.

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Abstract The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.

Official name

Oligonucleotides for inducing paternal UBE3A expression

Publication number

US12596118B2

Inventor / creator

Veronica Costa, Maj HEDTJÄRN, Marius Hoener, Ravi Jagasia, Mads Aaboe Jensen, Christoph Patsch, Lykke Pedersen, Søren Vestergaard Rasmussen

Assignee / owner

Hoffmann-La Roche Inc.

Filing

07/02/2025

Publication

04/07/2026

Expiry

07/02/2045

Status

ACTIVE

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