The Use of Small Molecules to Correct Defects in CFTR Folding, Maturation, and Channel Activity
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Cystic Fibrosis, one of the most common inherited lethal disease among Caucasians, is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. The CFTR protein acts as a gated Cl- channel at the apical membrane of epithelial ... ...
