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.
Claims13
A modified oligonucleotide comprising a contiguous nucleotide sequence of 13 to 30 nucleotides in length with nucleic acid complementarity to a contiguous portion of the nucleotide sequence set forth in SEQ ID NO:
- 2, wherein the modified oligonucleotide comprises from 5′ to 3′ (a) a first segment comprising 3-5 modified ribonucleotides, (b) a second segment comprising at least 7 deoxyribonucleotides, and (c) a third segment comprising 3-5 modified ribonucleotides.
Dependencies are inferred from the claim text.