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Article

Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice.

EMBO molecular medicine - 1 Jul 2013

Gedicke-Hornung Christina, Behrens-Gawlik Verena, Reischmann Silke, Geertz Birgit, Stimpel Doreen, Weinberger Florian, Schlossarek Saskia, Précigout Guillaume, Braren Ingke, Eschenhagen Thomas, Mearini Giulia, Lorain Stéphanie, Voit Thomas, Dreyfus Patrick A, Garcia Luis, Carrier Lucie

Abstract excerpt

Exon skipping mediated by antisense oligoribonucleotides (AON) is a promising therapeutic approach for genetic disorders, but has not yet been evaluated for cardiac diseases. We investigated the feasibility and efficacy of viral-mediated AON transfer in a Mybpc3-targeted knock-in (KI) mouse model...

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Share to Adenoviridae
Share to Cardiomyopathy, Hypertrophic

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Gene Knock-In Techniques

Share to Hypertrophy, Left Ventricular

Share to Myocardium

Share to RNA, Small Nuclear

Share to Transduction, Genetic

Source

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Identifiers

PMID: 23716398
DOI: 10.1002/emmm.201202168
PMCID: PMC3721478

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-23716398-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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