High Throughput Screening Identifies a Small Molecule Trafficking Corrector for Long-QT Syndrome Associated KCNQ1 Variants
Katherine R. Clowes, Carlos G. Vanoye, Ana Cristina Chang-Gonzalez, Ian M. Romaine, Katherine M. Stefanski, Mason C. Wilkinson, Joshua A. Bauer, Thomas P. Hasaka, Emily Days, Reshma Desai, Kathryn R. Butcher, Gary A. Sulikowksi, Alex G. Waterson, Jens Meiler, Kaitlyn V. Ledwitch, , Charles R. Sanders
This study identifies a small molecule, VU0494372, that enhances KCNQ1 expression and trafficking, offering a potential therapeutic approach for LQTS.
bioRxiv · Oct 13, 2025 · PreprintBiochemistry