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

• मुख्य लेखकों: Giulia Mearini (लेखक), Doreen Stimpel (लेखक), Elisabeth Krämer (लेखक), Birgit Geertz (लेखक), Ingke Braren (लेखक), Christina Gedicke-Hornung (लेखक), Guillaume Précigout (लेखक), Oliver J Müller (लेखक), Hugo A Katus (लेखक), Thomas Eschenhagen (लेखक), Thomas Voit (लेखक), Luis Garcia (लेखक), Stéphanie Lorain (लेखक), Lucie Carrier (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2013-01-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...