Angelman syndrome antisense treatment Patent uri icon

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.

author list (cited authors)

  • Dindot, S. V.

complete list of authors

  • Dindot, Scott Victor

publication date

  • December 2021