Correctors of ΔF508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein.
about
Pharmacological chaperoning: a primer on mechanism and pharmacologyCFTR Modulators: Shedding Light on Precision Medicine for Cystic FibrosisPrecision Genomic Medicine in Cystic FibrosisIncreasing the Endoplasmic Reticulum Pool of the F508del Allele of the Cystic Fibrosis Transmembrane Conductance Regulator Leads to Greater Folding Correction by Small Molecule TherapeuticsThe cystic fibrosis transmembrane conductance regulator (CFTR) and its stabilityClinical Pharmacogenetics Implementation Consortium (CPIC) guidelines for ivacaftor therapy in the context of CFTR genotypeSynergy-based small-molecule screen using a human lung epithelial cell line yields ΔF508-CFTR correctors that augment VX-809 maximal efficacy.Stabilizing rescued surface-localized δf508 CFTR by potentiation of its interaction with Na(+)/H(+) exchanger regulatory factor 1.A genomic signature approach to rescue ΔF508-cystic fibrosis transmembrane conductance regulator biosynthesis and function.VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1.Targeted proteomic quantitation of the absolute expression and turnover of cystic fibrosis transmembrane conductance regulator in the apical plasma membrane.Tgf-β1 inhibits Cftr biogenesis and prevents functional rescue of ΔF508-Cftr in primary differentiated human bronchial epithelial cells.CFTR and lung homeostasis.Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis.Discovery of novel potent ΔF508-CFTR correctors that target the nucleotide binding domain.PharmGKB summary: very important pharmacogene information for CFTRPharmacological correction of misfolding of ABC proteins.Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients.Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis.Molecular modelling and molecular dynamics of CFTR.Improved fluorescence assays to measure the defects associated with F508del-CFTR allow identification of new active compounds.Capturing the Direct Binding of CFTR Correctors to CFTR by Using Click Chemistry.A synonymous codon change alters the drug sensitivity of ΔF508 cystic fibrosis transmembrane conductance regulator.From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations.Restoration of NBD1 thermal stability is necessary and sufficient to correct ∆F508 CFTR folding and assemblyMechanism-based corrector combination restores ΔF508-CFTR folding and function.Progress towards next-generation therapeutics for cystic fibrosis.Repairing the basic defect in cystic fibrosis - one approach is not enough.Bone disease in cystic fibrosis: new pathogenic insights opening novel therapies.Orkambi® and amplifier co-therapy improves function from a rare CFTR mutation in gene-edited cells and patient tissue.Two Small Molecules Restore Stability to a Subpopulation of the Cystic Fibrosis Transmembrane Conductance Regulator with the Predominant Disease-causing Mutation.Efficacy of lumacaftor-ivacaftor for the treatment of cystic fibrosis patients homozygous for the F508del-CFTR mutation.Tools to explore ABCA3 mutations causing interstitial lung disease.The major cystic fibrosis causing mutation exhibits defective propensity for phosphorylation.From the endoplasmic reticulum to the plasma membrane: mechanisms of CFTR folding and trafficking.Current insights into the role of PKA phosphorylation in CFTR channel activity and the pharmacological rescue of cystic fibrosis disease-causing mutants.Increased efficacy of VX-809 in different cellular systems results from an early stabilization effect of F508del-CFTR.Chaperones rescue the energetic landscape of mutant CFTR at single molecule and in cell.Rescuing ΔF508 CFTR with trimethylangelicin, a dual-acting corrector and potentiator.Structural mechanisms of CFTR function and dysfunction.
P2860
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P2860
Correctors of ΔF508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein.
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
2012年论文
@zh
2012年论文
@zh-cn
name
Correctors of ΔF508 CFTR resto ...... tabilizing the mutant protein.
@ast
Correctors of ΔF508 CFTR resto ...... tabilizing the mutant protein.
@en
type
label
Correctors of ΔF508 CFTR resto ...... tabilizing the mutant protein.
@ast
Correctors of ΔF508 CFTR resto ...... tabilizing the mutant protein.
@en
prefLabel
Correctors of ΔF508 CFTR resto ...... tabilizing the mutant protein.
@ast
Correctors of ΔF508 CFTR resto ...... tabilizing the mutant protein.
@en
P2093
P2860
P356
P1433
P1476
Correctors of ΔF508 CFTR resto ...... tabilizing the mutant protein.
@en
P2093
Andrei A Aleksandrov
John R Riordan
Liying Cui
Pradeep Kota
Tim Jensen
P2860
P304
P356
10.1096/FJ.12-216119
P407
P577
2012-10-26T00:00:00Z