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Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice.
Gedicke-Hornung C, Behrens-Gawlik V, Reischmann S, Geertz B, Stimpel D, Weinberger F, Schlossarek S, Précigout G, Braren I, Eschenhagen T, Mearini G, Lorain S, Voit T, Dreyfus PA, Garcia L, Carrier L. Gedicke-Hornung C, et al. Among authors: stimpel d. EMBO Mol Med. 2013 Jul;5(7):1128-45. doi: 10.1002/emmm.201202168. Epub 2013 May 29. EMBO Mol Med. 2013. PMID: 23716398 Free PMC article.
Repair of Mybpc3 mRNA by 5'-trans-splicing in a Mouse Model of Hypertrophic Cardiomyopathy.
Mearini G, Stimpel D, Krämer E, Geertz B, Braren I, Gedicke-Hornung C, Précigout G, Müller OJ, Katus HA, Eschenhagen T, Voit T, Garcia L, Lorain S, Carrier L. Mearini G, et al. Among authors: stimpel d. Mol Ther Nucleic Acids. 2013 Jul 2;2(7):e102. doi: 10.1038/mtna.2013.31. Mol Ther Nucleic Acids. 2013. PMID: 23820890 Free PMC article.