These authors contributed equally to this work.
Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice
Version of Record online: 29 MAY 2013
Copyright © 2013 The Authors. Published by John Wiley and Sons, Ltd on behalf of EMBO
This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
EMBO Molecular Medicine
Volume 5, Issue 7, pages 1128–1145, July 2013
How to Cite
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, P. A., Garcia, L. and Carrier, L. (2013), Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice. EMBO Mol Med, 5: 1128–1145. doi: 10.1002/emmm.201202168
- Issue online: 3 JUL 2013
- Version of Record online: 29 MAY 2013
- Manuscript Revised: 19 APR 2013
- Manuscript Accepted: 19 APR 2013
- Manuscript Received: 19 OCT 2012
- Deutsche Forschungsgemeinschaft (FOR-604/1-2, CA 618/1-2)
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Figure S1. Evidence for an alternative Mybpc3 variant mRNA in wild-type mouse cardiac myocytes.
Figure S2. Evaluation of Var-4 Mybpc3 mRNA during mouse development.
Figure S3. Effect of 2OMePS-modified AONs on Mybpc3 mRNAs and cMyBP-C proteins in WT and KI cardiac.
Figure S4. Evaluation of the Mybpc3 mRNA species in wildtype cardiac myocytes.
Figure S5. Tissue distribution of GFP after systemic administration of AAV9 in a Mybpc3-targeted knock-in mouse.
Figure S6. Immunofluorescence analysis of cardiac sections after systemic administration of AAV9 in Mybpc3-targeted knock-in mice.
Figure S7. Variant-4 rescues the phenotype of Mybpc3-targeted KI mice in the heart in vivo.
Figure S8. Evaluation of AAV9 particles in ventricular tissue of KI injected mice.
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