GeneticsScience Translational MedicinePeer-reviewed
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…·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.
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