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

• Váldodahkkit: Giulia Mearini (Dahkki), Doreen Stimpel (Dahkki), Elisabeth Krämer (Dahkki), Birgit Geertz (Dahkki), Ingke Braren (Dahkki), Christina Gedicke-Hornung (Dahkki), Guillaume Précigout (Dahkki), Oliver J Müller (Dahkki), Hugo A Katus (Dahkki), Thomas Eschenhagen (Dahkki), Thomas Voit (Dahkki), Luis Garcia (Dahkki), Stéphanie Lorain (Dahkki), Lucie Carrier (Dahkki)
• Materiálatiipa: Girji
• Almmustuhtton: Elsevier, 2013-01-01T00:00:00Z.
• Fáttát: article
• Liŋkkat: Connect to this object online.

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...