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

• Main Authors: Giulia Mearini (Author), Doreen Stimpel (Author), Elisabeth Krämer (Author), Birgit Geertz (Author), Ingke Braren (Author), Christina Gedicke-Hornung (Author), Guillaume Précigout (Author), Oliver J Müller (Author), Hugo A Katus (Author), Thomas Eschenhagen (Author), Thomas Voit (Author), Luis Garcia (Author), Stéphanie Lorain (Author), Lucie Carrier (Author)
• Format: Book
• Published: Elsevier, 2013-01-01T00:00:00Z.
• Subjects: article
• Online Access: 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...