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Article

Suppression and Replacement Gene Therapy for KCNH2 -Mediated Arrhythmias

13 Oct 2022

Abstract excerpt

Background: KCNH2 -mediated arrhythmia syndromes are caused by loss-of-function (type 2 long QT syndrome [LQT2]) or gain-of-function (type 1 short QT syndrome [SQT1]) pathogenic variants in the KCNH2 -encoded K v 11.1 potassium channel, which is essential for the cardiac action potential. Methods: A dual-component “suppression-and-replacement” (SupRep) KCNH2 gene therapy was created by cloning into a single...

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PMID: 36252106
DOI: 10.1161/circgen.122.003719

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