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AAV9-cTnT-hPKG1
- Development stage
- Preclinical
- Lead developer
- University of Pittsburgh
- Modality
- Gene Therapies
- Administration
- Parenteral
01
Overview
AAV9-cTnT-hPKG1 is an adeno-associated virus (AAV) serotype 9-based gene therapy designed to selectively overexpress human protein kinase G 1 (PKG1) in cardiomyocytes. The construct utilizes a cardiac troponin T (cTnT) minimal promoter to ensure heart-specific expression of the hPKG1 transgene. This therapeutic approach aims to address functional natriuretic peptide (NP) deficiency and reduced myocardial cGMP levels, which are common features in heart failure. In preclinical murine models of dilated cardiomyopathy (DCM) and hypertrophic cardiomyopathy (HCM), AAV9-cTnT-hPKG1 has demonstrated the ability to rapidly improve left ventricular systolic and diastolic function, respectively, without significantly altering heart structure or causing hypertrophy.
02
Targets
PKG (cGMP-dependent protein kinase I alpha)
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