Open sesame! Compounds that correct the folding, trafficking and gating defects in cystic fibrosis transmembrane conductance regulator (CFTR), the main protein implicated in cystic fibrosis (CF), are of great interest as potential therapeutic agents for this lethal disease. The development of CFTR modulators, highlighting the main challenges in the field and advocating the application of rational rather than brute-force methods, is the focus of this Minireview.
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Noy et al. (2011) studied this question.
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