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

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 of...

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Detalles Bibliográficos
Autores principales:	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
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Blackwell Publishing Ltd 2013
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3721478/ https://www.ncbi.nlm.nih.gov/pubmed/23716398 http://dx.doi.org/10.1002/emmm.201202168

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