Mybpc3 gene therapy for neonatal cardiomyopathy enables long-term disease prevention in mice
Mybpc3 gene therapy for neonatal cardiomyopathy enables long-term disease prevention in mice
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Author / Creator
Mearini, Giulia , Stimpel, Doreen , Geertz, Birgit , Weinberger, Florian , Krämer, Elisabeth , Schlossarek, Saskia , Mourot-Filiatre, Julia , Stoehr, Andrea , Dutsch, Alexander , Wijnker, Paul J. M. , Braren, Ingke , Katus, Hugo A. , Müller, Oliver J. , Voit, Thomas , Eschenhagen, Thomas and Carrier, Lucie
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
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Publisher
London: Nature Publishing Group UK
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Scope and Contents
Contents
Homozygous or compound heterozygous frameshift mutations in
MYBPC3
encoding cardiac myosin-binding protein C (cMyBP-C) cause neonatal hypertrophic cardiomyopathy (HCM), which rapidly evolves into systolic heart failure and death within the first year of life. Here we show successful long-term
Mybpc3
gene therapy in homozygous
Mybpc3<...
Alternative Titles
Full title
Mybpc3 gene therapy for neonatal cardiomyopathy enables long-term disease prevention in mice
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TN_cdi_proquest_journals_1629415687
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_1629415687
Other Identifiers
ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/ncomms6515
