Article

CAG repeat-selective compounds reduce abundance of expanded CAG RNAs in patient cell and murine models of SCAs

2024-08-17

Abstract excerpt

<h4>Summary</h4> Spinocerebellar ataxias (SCAs) are a genetically heterogenous group of devastating neurodegenerative conditions for which clinical care currently focuses on managing symptoms. Across these diseases there is an unmet need for therapies that address underlying disease mechanisms. We utilised the shared CAG repeat expansion mutation causative for a large subgroup of SCAs, to develop a novel disease-...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 8b7f0514-3617-51f9-87a8-c39d4fe768a4
DOI: 10.1101/2024.08.17.608349

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

CAG repeat-selective compounds reduce abundance of expanded CAG RNAs in patient cell and murine models of SCAsDOI 10.1101/2024.08.17.608349Read

Widespread alternative splicing dysregulation occurs presymptomatically in CAG expansion spinocerebellar ataxias.Semantic proximity Read

Spinocerebellar ataxias caused by polyglutamine expansions: a review of therapeutic strategies.Semantic proximity Read

Recent therapeutic prospects for Machado–Joseph diseaseSemantic proximity Read

Metformin decreases RAN proteins, rescues splicing abnormalities and improves behavioral phenotypes in SCA8 BAC miceSemantic proximity Read

Increased FAN1 expression by mRNA-LNP attenuates CAG repeat expansion in Huntington patients’ iPSC-derived astrocytesSemantic proximity Read

A CTG repeat-selective chemical screen identifies microtubule inhibitors as selective modulators of toxic CUG RNA levelsSemantic proximity Read

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2.Semantic proximity Read

Interventionally targeting somatic CAG expansions can be a rapid disease-modifying therapeutic avenue: Preclinical evidenceSemantic proximity Read

A slipped-CAG DNA-binding small molecule induces trinucleotide-repeat contractions in vivo.Semantic proximity Read

Patterns of CAG repeat instability in the central nervous system and periphery in Huntington’s disease and in spinocerebellar ataxia type 1Semantic proximity Read

Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington's disease.Semantic proximity Read

Druggable genome screen identifies new regulators of the abundance and toxicity of ATXN3, the Spinocerebellar Ataxia Type 3 disease proteinSemantic proximity Read