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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...

Factors influencing readthrough therapy for frequent cystic fibrosis premature termination codons

Archive ouverte | Pranke, Iwona | CCSD

International audience. Premature termination codons (PTCs) are generally associated with severe forms of genetic diseases. Readthrough of in-frame PTCs using small molecules is a promising therapeutic approach. Non...

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...

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