Suppression-Replacement KCNQ1 Gene Therapy for Type 1 Long QT Syndrome
Abstract
Type 1 long QT syndrome (LQT1) is caused by loss-of-function variants in the KCNQ1-encoded Kv7.1 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.A dual-component suppression-and-replacement (SupRep) KCNQ1 gene therapy was created by cloning a KCNQ1 short hairpin RNA and a short hairpin RNA-immune KCNQ1 cDNA modified...
Paper Details
Title
Suppression-Replacement KCNQ1 Gene Therapy for Type 1 Long QT Syndrome
Published Date
Apr 6, 2021
Journal
Volume
143
Issue
14
Pages
1411 - 1425
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