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Article

Alpha protein kinase 3 gene therapy restores heart function in mouse and human models of cardiomyopathy

19 Aug 2026

Abstract excerpt

. Here we establish an adeno-associated virus (AAV)-mediated gene replacement therapy to deliver full-length human ALPK3. AAV-ALPK3 prevented disease in neonatal Alpk3-mutant mice and reversed established pathology in adults, with proteomic analysis demonstrating reversal of more than 95% of the molecular disease signature. Beyond ALPK3 deficiency, we explored broader therapeutic potential based on ALPK3's...

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PMID: 42618778
DOI: 10.1038/s44161-026-00843-1

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