Partial rescue of F508del‐cystic fibrosis transmembrane conductance regulator channel gating with modest improvement of protein processing, but not stability, by a dual‐acting small molecule
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DOI: 10.1111/bph.14141
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… read more here.
Keywords: protein processing; rescue f508del; channel gating;