Silencing the Mutant KCNH2 Allele to Reduce the Effects of Long QT Syndrome Type 2
Silencing of the mutant allele can substantially, but only partially, counteract the effects of mild or severe LQTS2 mutations on I<sub>Kr</sub>. Allele-specific inhibition of the mutant <i>KCNH2</i> allele alone is not sufficient to fully treat the effects of LQTS2 mutations and should be accompanied by a replacement gene therapy, creating a suppression-and-replacement ("SupRep") gene therapy.
Authors
- Ronald Wilders (ORCID: https://orcid.org/0000-0002-1340-0869)
Institutions
- Amsterdam University Medical Centers (NL)
- University of Amsterdam (NL)
Publication Details
- Journal
- Frontiers in Bioscience-Landmark
- Published
- 2026-02-12
- DOI
- https://doi.org/10.31083/fbl49097
- Primary Topic
- Cardiac electrophysiology and arrhythmias
- Type
- article
- Field-Weighted Citation Impact
- 0.00