Development of an ASO therapy for Angelman syndrome by targeting an evolutionarily conserved region at the start of the UBE3A-AS transcript
Scott V. Dindot, Sarah Christian, William J. Murphy, Allyson Berent, Jennifer Panagoulias, Annalise Schlafer, Johnathan L. Ballard, Kamelia Radeva, Ruth Robinson, Luke S. Myers, Thomas Jepp, Hillary Shaheen, Paul Hillman, Kranti Konganti, Andrew Hillhouse, , Jill L. Silverman
This paper describes the development of an antisense oligonucleotide (ASO) therapy targeting the UBE3A-AS transcript to reactivate paternal UBE3A expression in Angelman syndrome models.
Science Translational Medicine · Mar 22, 2023Peer-reviewedGenetics