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Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR
KTH, School of Engineering Sciences (SCI), Applied Physics. KTH, Centres, Science for Life Laboratory, SciLifeLab. Sorbonne Université, Muséum National d’Histoire Naturelle, UMR CNRS 7590, Institut de Minéralogie, de Physique des Matériaux et de Cosmochimie, IMPMC, Paris, 75005, France.ORCID iD: 0000-0002-5356-2440
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2021 (English)In: Cellular and Molecular Life Sciences (CMLS), ISSN 1420-682X, E-ISSN 1420-9071, Vol. 78, no 23, p. 7813-7829Article in journal (Refereed) Published
Abstract [en]

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 binding sites and mechanisms of action are still incompletely understood. Blind docking onto the 3D structures of both the first membrane-spanning domain (MSD1) and the first nucleotide-binding domain (NBD1), followed by molecular dynamics simulations, revealed the presence of two potential VX-809 corrector binding sites which, when mutated, abrogated rescue. Network of amino acids in the lasso helix 2 and the intracellular loops ICL1 and ICL4 allosterically coupled MSD1 and NBD1. Corrector VX-445 also occupied two potential binding sites on MSD1 and NBD1, the latter being shared with VX-809. Binding of both correctors on MSD1 enhanced the allostery between MSD1 and NBD1, hence the increased efficacy of the corrector combination. These correctors improve both intra-domain folding by stabilizing fragile protein–lipid interfaces and inter-domain assembly via distant allosteric couplings. These results provide novel mechanistic insights into the rescue of misfolded proteins by small molecules.

Place, publisher, year, edition, pages
Springer Nature , 2021. Vol. 78, no 23, p. 7813-7829
Keywords [en]
Binding site, Chemical chaperone, Cystic fibrosis, Molecular dynamics, Protein folding
National Category
Microbiology
Identifiers
URN: urn:nbn:se:kth:diva-312840DOI: 10.1007/s00018-021-03994-5ISI: 000712777000003PubMedID: 34714360Scopus ID: 2-s2.0-85118259047OAI: oai:DiVA.org:kth-312840DiVA, id: diva2:1660404
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QC 20220524

Available from: 2022-05-24 Created: 2022-05-24 Last updated: 2022-06-25Bibliographically approved

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Elbahnsi, Ahmad

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