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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Main Authors: Giulia Mearini, Doreen Stimpel, Elisabeth Krämer, Birgit Geertz, Ingke Braren, Christina Gedicke-Hornung, Guillaume Précigout, Oliver J Müller, Hugo A Katus, Thomas Eschenhagen, Thomas Voit, Luis Garcia, Stéphanie Lorain, Lucie Carrier
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2013-01-01
Colecção:Molecular Therapy: Nucleic Acids
Assuntos:
hypertrophic cardiomyopathy
Mybpc3
RNA-based therapy
trans-splicing
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S2162253116301639
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