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US 11,685,920 B2Granted
Angelman syndrome antisense treatment
AssigneeThe Texas A&M University System
InventorScott Victor Dindot
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Abstract
Disclosed herein are antisense 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 at the 5′-end of UBE3A-AS, which is downstream of SNORD115-45 snoRNA. Also disclosed are pharmaceutical compositions and methods for treatment of Angelman syndrome.
Claims1
An antisense oligonucleotide comprising the nucleotide sequence AL*GL*AL*a*t*g*g*c*a*c*a*t*c*t*5mCL*TL*TL*GL (SEQ ID NO:
- 381), wherein:
- capital letters denote RNA nucleosides;
- lower case letters denote DNA nucleosides;
- 5mC denotes a 5-methylcytosine nucleoside;
- superscript L denotes a locked nucleic acid (LNA) nucleoside;
- and
- asterisks denote phosphorothioate internucleoside linkages.
4 more elements
Dependencies are inferred from the claim text.