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.
Claims16
1-13. (canceled)
A method for treating or preventing Angelman syndrome in a subject comprising administering to a subject suffering from or susceptible to Angelman syndrome a therapeutically or prophylactically effective amount a pharmaceutical composition comprising an antisense oligonucleotide consisting of a nucleotide sequence 10 to 30 nucleotides in length having a nucleic acid sequence complementary to a contiguous portion of SEQ ID NO:
- 2.
Dependencies are inferred from the claim text.