<scp>CFTR</scp> potentiators partially restore channel function to <scp>A</scp>561<scp>E</scp>‐CFTR, a cystic fibrosis mutant with a similar mechanism of dysfunction as <scp>F</scp>508del‐<scp>CFTR</scp>

Abstract

Like F508del-CFTR, A561E-CFTR perturbs protein processing, thermostability and channel gating. CFTR potentiators partially restore channel function to low temperature-rescued A561E-CFTR. Transformational drug therapy for A561E-CFTR is likely to require CFTR correctors, CFTR potentiators and special attention to thermostability.