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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Detaylı Bibliyografya
Yayımlandı:JCI Insight
Asıl Yazarlar: Li, Jiayang, Mamidi, Ranganath, Doh, Chang Yoon, Holmes, Joshua B., Bharambe, Nikhil, Ramachandran, Rajesh, Stelzer, Julian E.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: American Society for Clinical Investigation 2020
Konular:
Research Article
Online Erişim: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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