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Article

Suppression-Replacement KCNQ1 Gene Therapy for Type 1 Long QT Syndrome

28 Jan 2021

Abstract excerpt

Background: Type 1 long QT syndrome (LQT1) is caused by loss-of-function variants in the KCNQ1 -encoded K v 7.1 potassium channel α-subunit that is essential for cardiac repolarization, providing the slow delayed rectifier current. No current therapies target the molecular cause of LQT1. Methods: A dual-component suppression-and-replacement (SupRep) KCNQ1 gene therapy was created by cloning a KCNQ1 short hairpin...

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PMID: 33504163
DOI: 10.1161/circulationaha.120.051836

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