Article

Alpha Protein Kinase 3 Gene Therapy Restores Heart Function in Mouse and Human Models of Cardiomyopathy

2025-08-02

Abstract excerpt

Truncating variants in the Alpha Kinase 3 (ALPK3) gene have recently emerged as an important cause of genetic cardiomyopathy globally and here we demonstrate the efficacy and safety of a viral-based gene replacement therapy for ALPK3. Given the loss-of-function nature of these variants, we reasoned that a gene replacement approach would improve heart function in this patient population. We demonstrate that the del...

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Identifiers and source

Literature Corpus work: 7422097d-9b94-50f3-8aac-3758cd011b97
DOI: 10.1101/2025.07.31.667858

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