Rescue of F508del‐cystic fibrosis (CF) transmembrane conductance regulator (CFTR), the most common CF mutation, requires small molecules that overcome protein processing, stability and channel gating defects. Here, we investigate F508del‐CFTR… Click to show full abstract
Rescue of F508del‐cystic fibrosis (CF) transmembrane conductance regulator (CFTR), the most common CF mutation, requires small molecules that overcome protein processing, stability and channel gating defects. Here, we investigate F508del‐CFTR rescue by CFFT‐004, a small molecule designed to independently correct protein processing and channel gating defects.
               
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