In vivo rescue of genetic dilated cardiomyopathy by systemic delivery of nexilin.

ABSTRACT:

Background

Dilated cardiomyopathy (DCM) is one of the most common causes of heart failure. Multiple identified mutations in nexilin (NEXN) have been suggested to be linked with severe DCM. However, the exact association between multiple mutations of Nexn and DCM remains unclear. Moreover, it is critical for the development of precise and effective therapeutics in treatments of DCM.

Results

In our study, Nexn global knockout mice and mice carrying human equivalent G645del mutation are studied using functional gene rescue assays. AAV-mediated gene delivery is conducted through systemic intravenous injections at the neonatal stage. Heart tissues are analyzed by immunoblots, and functions are assessed by echocardiography. Here, we identify functional components of Nexilin and de

SUBMITTER: Shao Y 

PROVIDER: S-EPMC11112773 | biostudies-literature | 2024 May

REPOSITORIES: biostudies-literature