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

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

Abstract 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...

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Main Authors: Christina Gedicke‐Hornung, Verena Behrens‐Gawlik, Silke Reischmann, Birgit Geertz, Doreen Stimpel, Florian Weinberger, Saskia Schlossarek, Guillaume Précigout, Ingke Braren, Thomas Eschenhagen, Giulia Mearini, Stéphanie Lorain, Thomas Voit, Patrick A. Dreyfus, Luis Garcia, Lucie Carrier
Format: Article
Language:English
Published: Springer Nature 2013-05-01
Series:EMBO Molecular Medicine
Subjects:
alternative splicing
antisense oligoribonucleotide
cardiac myosin‐binding protein‐C
exon skipping
hypertrophic cardiomyopathy
Online Access:https://doi.org/10.1002/emmm.201202168
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https://doi.org/10.1002/emmm.201202168

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