Repair of Mybpc3 mRNA by 5′-trans-splicing in a Mouse Model of Hypertrophic Cardiomyopathy

RNA trans-splicing has been explored as a therapeutic option for a variety of genetic diseases, but not for cardiac genetic disease. Hypertrophic cardiomyopathy (HCM) is an autosomal-dominant disease, characterized by left ventricular hypertrophy (LVH) and diastolic dysfunction. MYBPC3, encoding car...

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Detalhes bibliográficos
Main Authors: Mearini, Giulia, Stimpel, Doreen, Krämer, Elisabeth, Geertz, Birgit, Braren, Ingke, Gedicke-Hornung, Christina, Précigout, Guillaume, Müller, Oliver J, Katus, Hugo A, Eschenhagen, Thomas, Voit, Thomas, Garcia, Luis, Lorain, Stéphanie, Carrier, Lucie
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2013
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3731888/
https://ncbi.nlm.nih.gov/pubmed/23820890
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2013.31
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