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Related Experiment Video

Updated: Jul 15, 2026

Behavioral Characterization of an Angelman Syndrome Mouse Model
11:05

Behavioral Characterization of an Angelman Syndrome Mouse Model

Published on: October 20, 2023

Emerging Therapies for Angelman Syndrome.

Cyril Tychon1, Theodora Markati2,3, Serpil Alkan1,4

  • 1Department of Pediatrics, CHU de Liège-CHR de la Citadelle, University of Liège, 4000, Liège, Belgium.

CNS Drugs
|July 14, 2026
PubMed
Summary

Angelman syndrome treatments are advancing by targeting the UBE3A-antisense transcript (UBE3A-ATS) to restore UBE3A protein. Early intervention strategies, including antisense oligonucleotides, show promise for improving outcomes in this neurogenetic disorder.

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Area of Science:

  • Neurogenetics
  • Molecular Biology
  • Developmental Disorders

Background:

  • Angelman syndrome (AS) is a severe neurogenetic disorder caused by deficient neuronal expression of the UBE3A protein.
  • Current AS management is primarily symptomatic, highlighting the need for disease-modifying therapies.

Purpose of the Study:

  • To review emerging therapeutic strategies for Angelman syndrome focused on restoring UBE3A function.
  • To discuss the potential of targeting the UBE3A-antisense transcript (UBE3A-ATS) to unsilence the paternal UBE3A allele.

Main Methods:

  • Review of current and emerging therapeutic modalities for AS, including gene replacement and allele unsilencing.
  • Focus on antisense oligonucleotides (ASOs) and other approaches targeting the UBE3A-ATS.

Main Results:

  • Several antisense oligonucleotide (ASO) programs targeting UBE3A-ATS have shown promising early clinical results.
  • Improvements in clinical outcomes and EEG biomarkers have been reported in early-stage AS trials.

Conclusions:

  • Targeting UBE3A-ATS represents a promising therapeutic avenue for Angelman syndrome.
  • Early intervention and newborn screening are being explored to improve long-term outcomes for individuals with AS.