Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice. [electronic resource]
Producer: 20131029Description: 1128-45 p. digitalISSN:- 1757-4684
- Adenoviridae -- genetics
- Alternative Splicing
- Animals
- Cardiomyopathy, Hypertrophic -- genetics
- Carrier Proteins -- genetics
- Exons
- Gene Knock-In Techniques
- Genetic Therapy
- HEK293 Cells
- Heart -- physiopathology
- Humans
- Hypertrophy, Left Ventricular -- prevention & control
- Mice
- Mutation
- Myocardium -- metabolism
- Oligoribonucleotides, Antisense -- administration & dosage
- Protein Isoforms -- genetics
- RNA, Messenger -- genetics
- RNA, Small Nuclear -- administration & dosage
- Transduction, Genetic
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Publication Type: Journal Article; Research Support, Non-U.S. Gov't
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