ArticleThe Biochemical journal2006
The chemical chaperone CFcor-325 repairs folding defects in the transmembrane domains of CFTR-processing mutants.
Article in The Biochemical journal, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed, 45 citations in OpenAlex.
- Clinical and genetic features of cystic fibrosis in Japan.Journal of human genetics · 2023Article
- Targeting trafficking as a therapeutic avenue for misfolded GPCRs leading to endocrine diseases.Frontiers in endocrinology · 2022Review
- Residual function of cystic fibrosis mutants predicts response to small molecule CFTR modulators.JCI insight · 2018Article
- Repairing folding-defective α-sarcoglycan mutants by CFTR correctors, a potential therapy for limb-girdle muscular dystrophy 2D.Human molecular genetics · 2018Article
- Article
- Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives.Clinical pharmacology : advances and applications · 2016Review
- Increased efficacy of VX-809 in different cellular systems results from an early stabilization effect of F508del-CFTR.Pharmacology research & perspectives · 2015Article
- Anchored PDE4 regulates chloride conductance in wild-type and ΔF508-CFTR human airway epithelia.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2014Article
- Functional Rescue of F508del-CFTR Using Small Molecule Correctors.Frontiers in pharmacology · 2012Article
- Ouabain Mimics Low Temperature Rescue of F508del-CFTR in Cystic Fibrosis Epithelial Cells.Frontiers in pharmacology · 2012Article
- Thermally unstable gating of the most common cystic fibrosis mutant channel (ΔF508): "rescue" by suppressor mutations in nucleotide binding domain 1 and by constitutive mutations in the cytosolic loops.The Journal of biological chemistry · 2011Article
- The V510D suppressor mutation stabilizes DeltaF508-CFTR at the cell surface.Biochemistry · 2010Article
- DeltaF508 CFTR processing correction and activity in polarized airway and non-airway cell monolayers.Pulmonary pharmacology & therapeutics · 2010Article
- Modulation of endocytic trafficking and apical stability of CFTR in primary human airway epithelial cultures.American journal of physiology. Lung cellular and molecular physiology · 2010Article
- The Use of Small Molecules to Correct Defects in CFTR Folding, Maturation, and Channel Activity.Current chemical biology · 2009Article
- Chemical and biological folding contribute to temperature-sensitive DeltaF508 CFTR trafficking.Traffic (Copenhagen, Denmark) · 2008Article
- Enhanced cell-surface stability of rescued DeltaF508 cystic fibrosis transmembrane conductance regulator (CFTR) by pharmacological chaperones.The Biochemical journal · 2008Article
- The role of the UPS in cystic fibrosis.BMC biochemistry · 2007Review
- Isofagomine- and 2,5-anhydro-2,5-imino-D-glucitol-based glucocerebrosidase pharmacological chaperones for Gaucher disease intervention.Journal of medicinal chemistry · 2007Article
- Using a cysteine-less mutant to provide insight into the structure and mechanism of CFTR.The Journal of physiology · 2006Article
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Most patients with CF (cystic fibrosis) express a CFTR [CF TM (transmembrane) conductance regulator] processing mutant that is not trafficked to the cell surface because it is retained in the endoplasmic reticulum due to altered packing of the TM segments. CL4 (cytoplasmic loop 4) connecting TMs 10 and 11 is a 'hot-spot' for CFTR processing mutations. The chemical chaperone CFcor-325 (4-cyclohexyloxy-2-{1-[4-(4-methoxy-benezenesulphonyl)piperazin-1-yl]-ethyl}-quinazoline) rescued most CL4 mutants. To test if CFcor-325 promoted correct folding of the TMDs (TM domains), we selected two of the CL4 mutants (Q1071P and H1085R) for disulphide cross-linking analysis. Pairs of cysteine residues that were cross-linked in mature wild-type CFTR were introduced into mutants Q1071P and H1085R. The cross-linking patterns of the Q1071P or H1085R double cysteine mutants rescued with CFcor-325 were similar to those observed with mature wild-type double cysteine proteins. These results show that CFcor-325 rescued CFTR mutants by repairing the folding defects in the TMDs.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.