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

• Asıl Yazarlar: Giulia Mearini (Yazar), Doreen Stimpel (Yazar), Elisabeth Krämer (Yazar), Birgit Geertz (Yazar), Ingke Braren (Yazar), Christina Gedicke-Hornung (Yazar), Guillaume Précigout (Yazar), Oliver J Müller (Yazar), Hugo A Katus (Yazar), Thomas Eschenhagen (Yazar), Thomas Voit (Yazar), Luis Garcia (Yazar), Stéphanie Lorain (Yazar), Lucie Carrier (Yazar)
• Materyal Türü: Kitap
• Baskı/Yayın Bilgisi: Elsevier, 2013-01-01T00:00:00Z.
• Konular: article
• Online Erişim: 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...