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Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR

Archive ouverte | Baatallah, Nesrine | CCSD

International audience. Protein misfolding is involved in a large number of diseases, among which cystic fibrosis. Complex intra- and inter-domain folding defects associated with mutations in the cystic fibrosis tra...

Cis variants identified in F508del complex alleles modulate CFTR channel rescue by small molecules

Archive ouverte | Baatallah, Nesrine | CCSD

International audience

New insights into structure and function of bis-phosphinic acid derivatives and implications for CFTR modulation

Archive ouverte | Bitam, Sara | CCSD

International audience. C407 is a compound that corrects the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein carrying the p.Phe508del (F508del) mutation. We investigated the corrector effect of c4...

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