The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Mar. 25, 2025

Filed:

Feb. 22, 2023
Applicant:

Hoffmann-la Roche Inc., Little Falls, NJ (US);

Inventors:

Veronica Costa, Basel, CH;

Maj Hedtjärn, Hørsholm, DK;

Marius Hoener, Basel, CH;

Ravi Jagasia, Basel, CH;

Mads Aaboe Jensen, Hørsholm, DK;

Christoph Patsch, Basel, CH;

Lykke Pedersen, Hørsholm, DK;

Søren Vestergaard Rasmussen, Hørsholm, DK;

Assignee:

Hoffmann-La Roche Inc., Little Falls, NJ (US);

Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/11 (2006.01); C12N 5/00 (2006.01); C12N 5/074 (2010.01); C12N 5/0793 (2010.01); C12N 5/0797 (2010.01); C12N 15/113 (2010.01); G01N 33/50 (2006.01);
U.S. Cl.
CPC ...
G01N 33/5014 (2013.01); C12N 5/0018 (2013.01); C12N 5/0619 (2013.01); C12N 5/0623 (2013.01); C12N 5/0696 (2013.01); C12N 15/11 (2013.01); C12N 15/111 (2013.01); C12N 15/113 (2013.01); C12N 15/1137 (2013.01); G01N 33/5058 (2013.01); C12N 2310/11 (2013.01); C12N 2310/20 (2017.05); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/33 (2013.01); C12N 2310/3341 (2013.01); C12N 2310/341 (2013.01); C12N 2310/346 (2013.01); C12N 2310/351 (2013.01); C12N 2320/34 (2013.01); C12N 2500/38 (2013.01); C12N 2500/44 (2013.01); C12N 2501/01 (2013.01); C12N 2501/11 (2013.01); C12N 2501/115 (2013.01); C12N 2501/119 (2013.01); C12N 2501/13 (2013.01); C12N 2501/41 (2013.01); C12N 2501/60 (2013.01); C12N 2501/727 (2013.01); C12N 2501/999 (2013.01); C12N 2503/02 (2013.01); C12N 2506/45 (2013.01); C12N 2533/52 (2013.01);
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 SNORD1091B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.


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