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Article

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

19 Jul 2025

Abstract excerpt

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...

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PMID: 40683992
DOI: 10.1038/s41434-025-00553-6

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