High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy

The selection of an appropriate promoter is important to the design and optimisation of adeno-associated viral (AAV) vector-based cardiac gene therapies. The expression cassette design can impact efficacy and safety of the vector. This study is the first to use a novel AAV barcode-seq method for the simultaneous evaluation of a panel of cardiac-specific promoters in a high-throughput manner. Functional analyses of our cardiac promoter kit packaged in three different capsids were performed using neonatal rat ventricular myocytes (NRVM), human iPSC-derived cardiomyocytes (hiPSC-CMs), HuH7 hepatocellular carcinoma cells, as well as mouse, rat, sheep and pig models. The cardiac troponin T (cTnT) promoter showed the most promise overall as a cardiac-specific promoter across all cardiac models tested. The results validate the barcode-seq technique as a powerful and versatile approach that enables high-throughput, quantitative analysis of various expression cassettes in commonly used models of cardiac gene therapy.

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Publication Details

Journal
Gene Therapy
Published
2025-07-19
DOI
https://doi.org/10.1038/s41434-025-00553-6
Citations
2
Primary Topic
Viral Infections and Immunology Research
Type
article
Field-Weighted Citation Impact
2.78

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High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy

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High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy

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article en
2 citations

Abstract

The selection of an appropriate promoter is important to the design and optimisation of adeno-associated viral (AAV) vector-based cardiac gene therapies. The expression cassette design can impact efficacy and safety of the vector. This study is the first to use a novel AAV barcode-seq method for the simultaneous evaluation of a panel of cardiac-specific promoters in a high-throughput manner. Functional analyses of our cardiac promoter kit packaged in three different capsids were performed using neonatal rat ventricular myocytes (NRVM), human iPSC-derived cardiomyocytes (hiPSC-CMs), HuH7 hepatocellular carcinoma cells, as well as mouse, rat, sheep and pig models. The cardiac troponin T (cTnT) promoter showed the most promise overall as a cardiac-specific promoter across all cardiac models tested. The results validate the barcode-seq technique as a powerful and versatile approach that enables high-throughput, quantitative analysis of various expression cassettes in commonly used models of cardiac gene therapy.

Gene Therapy
The University of Sydney (AU), Children's Medical Research Institute (AU), Children's Hospital at Westmead (AU), Westmead Hospital (AU), Westmead Institute for Medical Research (AU), Sydney Children’s Hospitals Network (AU), Narodowy Instytut Leków (PL)
National Health and Medical Research Council
Good health and well-being
Openalex Percentile: Top 9%
Viral Infections and Immunology Research
2.78
DOIOpen Access PDFOpenAlex
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