A new corrector rescues F508del-CFTR folding through stabilization of the TMD1-NBD1 linker
Peter van der Sluijs, Olga Papaioannou, Azib Hagos, Aya M. Saleh, Timothy R. Hodges, Corina Balut, Matthew Green, Sara Alani, Yihong Fan, Kazumi Ebine, Michael R. Schrimpf, David J. Hardee, Huan-Qiu Li, Henry Korman, Laura Tade, , Ineke Braakman
This paper introduces X307810, a new C2 corrector that significantly improves F508del-CFTR folding and function by stabilizing the TMD1-NBD1 linker, achieving 80% wild-type levels.
bioRxiv · Mar 10, 2025 · PreprintBiochemistry