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Article

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

1 Jan 2013

Abstract excerpt

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 cardiac myosin-binding protein C (cMyBP-C) is frequently mutated. We evaluated the 5'-trans-splicing strategy in a...

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PMID: 23820890
DOI: 10.1038/mtna.2013.31

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Repair of Mybpc3 mRNA by 5′-trans-splicing in a Mouse Model of Hypertrophic CardiomyopathyPMID 23820890Read ↗

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