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

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

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Detalles Bibliográficos
Autores principales: Cócera-Ortega, Lucía, Wilders, Ronald, Kamps, Selina C., Fabrizi, Benedetta, Huber, Irit, van der Made, Ingeborg, van den Bout, Anouk, de Vries, Dylan K., Gepstein, Lior, Verkerk, Arie O., Pinto, Yigal M., Tijsen, Anke J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9000197/
https://www.ncbi.nlm.nih.gov/pubmed/35409410
http://dx.doi.org/10.3390/ijms23074053

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