shRNAs Targeting a Common <i>KCNQ1</i> Variant Could Alleviate Long-QT1 Disease Severity by Inhibiting a Mutant Allele

shRNAs Targeting a Common <i>KCNQ1</i> Variant Could Alleviate Long-QT1 Disease Severity by Inhibiting a Mutant Allele

Long-QT syndrome type 1 (LQT1) is caused by mutations in <i>KCNQ1</i>. Patients heterozygous for such a mutation co-assemble both mutant and wild-type <i>KCNQ1</i>-encoded subunits into tetrameric Kv7.1 potassium channels. Here, we investigated whether allele-specific inhibit...

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Bibliographic Details
Main Authors: Lucía Cócera-Ortega, Ronald Wilders, Selina C. Kamps, Benedetta Fabrizi, Irit Huber, Ingeborg van der Made, Anouk van den Bout, Dylan K. de Vries, Lior Gepstein, Arie O. Verkerk, Yigal M. Pinto, Anke J. Tijsen
Format: Article
Language:English
Published: MDPI AG 2022-04-01
Series:International Journal of Molecular Sciences
Subjects:
long-QT syndrome type 1
hiPSC-cardiomyocytes
RNA interference
gene therapy
arrhythmia
Online Access:https://www.mdpi.com/1422-0067/23/7/4053

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