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

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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Bibliographic Details
Main Authors: Jiayang Li, Ranganath Mamidi, Chang Yoon Doh, Joshua B. Holmes, Nikhil Bharambe, Rajesh Ramachandran, Julian E. Stelzer
Format: Article
Language:English
Published: American Society for Clinical investigation 2020-09-01
Series:JCI Insight
Subjects:
Cardiology
Online Access:https://doi.org/10.1172/jci.insight.130182

Internet

https://doi.org/10.1172/jci.insight.130182

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