Oligonucleotides for inducing paternal UBE3A expression

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专利权人:

ROCHE INNOVATION CENTER COPENHAGEN A/S;HOFFMANN-LA ROCHE INC.

发明人:

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

申请号：

US16388714

公开号：

US10739332B2

申请日:

2019.04.18

申请国别(地区):

US

年份:

2020

代理人:

摘要：

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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