Angelman syndrome antisense treatment
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.
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Citation
Dindot, Scott Victor (2021). Angelman syndrome antisense treatment. United States. Patent and Trademark Office; Texas A&M University. Libraries. Available electronically from https : / /hdl .handle .net /1969 .1 /195043.