Article
Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR.
Cellular and molecular life sciences : CMLS - 1 Dec 2021
Baatallah Nesrine, Elbahnsi Ahmad, Mornon Jean-Paul, Chevalier Benoit, Pranke Iwona, Servel Nathalie, Zelli Renaud, Décout Jean-Luc, Edelman Aleksander, Sermet-Gaudelus Isabelle, Callebaut Isabelle, Hinzpeter Alexandre
Abstract excerpt
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 transmembrane regulator (CFTR) gene, among which p.Phe508del (F508del), have recently become a therapeutical target. Clinically approved correctors such as VX-809, VX-661, and VX-445, rescue mutant protein. However, their...
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