Related Experiment Video
Updated: Jul 15, 2026
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
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.
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.
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