CFTR corrector efficacy is associated with occupancy of distinct binding sites
Nesrine Baatallah, Ahmad Elbahnsi, Jean‐Paul Mornon, B. Chevalier, Iwona Maria Pranke, Nathalie Servel, Renaud Zelli, Jean‐Luc Décout, Aleksander Edelman, Isabelle Sermet‐Gaudelus, Isabelle Callebaut, Alexandre Hinzpeter
The paper identifies two distinct VX-809 binding sites on CFTR that are critical for corrector efficacy, showing different sensitivity to mutations and allostery.
bioRxiv · May 4, 2021 · PreprintMolecular Biology