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KCNQ1 suppression-replacement gene therapy in transgenic rabbits with type 1 long QT syndrome

8 Aug 2024

Abstract excerpt

BACKGROUND AND AIMS: Type 1 long QT syndrome (LQT1) is caused by pathogenic variants in the KCNQ1-encoded Kv7.1 potassium channels, which pathologically prolong ventricular action potential duration (APD). Herein, the pathologic phenotype in transgenic LQT1 rabbits is rescued using a novel KCNQ1 suppression-replacement (SupRep) gene therapy. METHODS: KCNQ1-SupRep gene therapy was developed by combining into a...

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PMID: 39115049
DOI: 10.1093/eurheartj/ehae476

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