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
An antisense oligonucleotide comprising a contiguous nucleotide sequence of 10 to 30 nucleotides in length with at least 98% complementarity to a contiguous portion of nucleic acid sequence SEQ ID NO:1, SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, or SEQ ID NO:5.
Dependencies are inferred from the claim text.