AAV9 gene transfer of cMyBPC N-terminal domains ameliorates cardiomyopathy in cMyBPC-deficient mice

Decreased cardiac myosin-binding protein C (cMyBPC) expression due to inheritable mutations is thought to contribute to the hypertrophic cardiomyopathy (HCM) phenotype, suggesting that increasing cMyBPC content is of therapeutic benefit. In vitro assays show that cMyBPC N-terminal domains (NTDs) con...

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Pubblicato in:JCI Insight
Autori principali: Li, Jiayang, Mamidi, Ranganath, Doh, Chang Yoon, Holmes, Joshua B., Bharambe, Nikhil, Ramachandran, Rajesh, Stelzer, Julian E.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society for Clinical Investigation 2020
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7526450/
https://ncbi.nlm.nih.gov/pubmed/32750038
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.130182
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