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Ube3a unsilencer for the potential treatment of Angelman syndrome

8 Jul 2024

Abstract excerpt

Deletion of the maternal UBE3A allele causes Angelman syndrome (AS); because paternal UBE3A is epigenetically silenced by a long non-coding antisense (UBE3A-ATS) in neurons, this nearly eliminates UBE3A protein in the brain. Reactivating paternal UBE3A holds promise for treating AS. We previously showed topoisomerase inhibitors can reactivate paternal UBE3A, but their therapeutic challenges prompted our search...

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Share to Epigenetics and DNA Methylation
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PMID: 38977672
DOI: 10.1038/s41467-024-49788-8

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

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Ube3a unsilencer for the potential treatment of Angelman syndromePMID 38977672Read ↗

Towards a therapy for Angelman syndrome by targeting a long non-coding RNA.Semantic proximity Read ↗

Development of an ASO therapy for Angelman syndrome by targeting an evolutionarily conserved region at the start of the <i>UBE3A-AS</i> transcriptSemantic proximity Read ↗

Topoisomerase inhibitors unsilence the dormant allele of Ube3a in neurons.Semantic proximity Read ↗

An ASO therapy for Angelman syndrome that targets an evolutionarily conserved region at the start of the UBE3A-AS transcript.Semantic proximity Read ↗

Emerging Gene and Small Molecule Therapies for the Neurodevelopmental Disorder Angelman SyndromeSemantic proximity Read ↗

Antisense oligonucleotides targeting <i>UBE3A-ATS</i> restore expression of <i>UBE3A</i> by relieving transcriptional interferenceSemantic proximity Read ↗

UBE3A reinstatement as a disease‐modifying therapy for Angelman syndromeSemantic proximity Read ↗

Therapies in preclinical and clinical development for Angelman syndromeSemantic proximity Read ↗

CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in miceSemantic proximity Read ↗

Truncation of Ube3a-ATS Unsilences Paternal Ube3a and Ameliorates Behavioral Defects in the Angelman Syndrome Mouse ModelSemantic proximity Read ↗

The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trialSemantic proximity Read ↗

Ube3a-ATS is an atypical RNA polymerase II transcript that represses the paternal expression of Ube3aSemantic proximity Read ↗

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