相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。Mechanism of CFTR correction by type I folding correctors
Karol Fiedorczuk et al.
CELL (2022)
Rare Trafficking CFTR Mutations Involve Distinct Cellular Retention Machineries and Require Different Rescuing Strategies
Sofia S. Ramalho et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2022)
Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial
Sivagurunathan Sutharsan et al.
LANCET RESPIRATORY MEDICINE (2022)
Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516A
Miqueias Lopes-Pacheco et al.
CELLS (2022)
Can two wrongs make a right? F508del-CFTR ion channel rescue by second-site mutations in its transmembrane domains
Stella Prins et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2022)
The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis
Enrico Costa et al.
ORPHANET JOURNAL OF RARE DISEASES (2022)
Advances in Preclinical In Vitro Models for the Translation of Precision Medicine for Cystic Fibrosis
Iris A. L. Silva et al.
JOURNAL OF PERSONALIZED MEDICINE (2022)
Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR
Nesrine Baatallah et al.
CELLULAR AND MOLECULAR LIFE SCIENCES (2021)
Discovery of CFTR modulators for the treatment of cystic fibrosis
Miqueias Lopes-Pacheco et al.
EXPERT OPINION ON DRUG DISCOVERY (2021)
Partial Rescue of F508del-CFTR Stability and Trafficking Defects by Double Corrector Treatment
Valeria Capurro et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)
Personalized Medicine Based on Nasal Epithelial Cells: Comparative Studies with Rectal Biopsies and Intestinal Organoids
Iris A. L. Silva et al.
JOURNAL OF PERSONALIZED MEDICINE (2021)
A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants
Guido Veit et al.
JOURNAL OF PERSONALIZED MEDICINE (2021)
Biological Characterization of F508delCFTR Protein Processing by the CFTR Corrector ABBV-2222/GLPG2222
Ashvani K. Singh et al.
JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2020)
CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine
Miqueias Lopes-Pacheco
FRONTIERS IN PHARMACOLOGY (2020)
ProteinsPlus: interactive analysis of protein-ligand binding interfaces
Katrin Schoening-Stierand et al.
NUCLEIC ACIDS RESEARCH (2020)
Characterization of the mechanism of action of RDR01752, a novel corrector of F508del-CFTR
Miqueias Lopes-Pacheco et al.
BIOCHEMICAL PHARMACOLOGY (2020)
Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination
Guido Veit et al.
JCI INSIGHT (2020)
Design, synthesis, structural characterization and in vitro evaluation of new 1,4-disubstituted-1,2,3-triazole derivatives against glioblastoma cells
Veronica D. da Silva et al.
BIOORGANIC CHEMISTRY (2019)
Structural identification of a hotspot on CFTR for potentiation
Fangyu Liu et al.
SCIENCE (2019)
Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
P. G. Middleton et al.
NEW ENGLAND JOURNAL OF MEDICINE (2019)
Structure-guided combination therapy to potently improve the function of mutant CFTRs
Guido Veit et al.
NATURE MEDICINE (2018)
Direct Binding of the Corrector VX-809 to Human CFTR NBD1: Evidence of an Allosteric Coupling between the Binding Site and the NBD1:CL4 Interface
Rhea P. Hudson et al.
MOLECULAR PHARMACOLOGY (2017)
ProteinsPlus: a web portal for structure analysis of macromolecules
Rainer Faehrrolfes et al.
NUCLEIC ACIDS RESEARCH (2017)
SwissADME: a free web tool to evaluate pharmacokinetics, drug-likeness and medicinal chemistry friendliness of small molecules
Antoine Daina et al.
SCIENTIFIC REPORTS (2017)
The 1,2,3-triazole ring as a bioisostere in medicinal chemistry
Elisa Bonandi et al.
DRUG DISCOVERY TODAY (2017)
Combination of Correctors Rescues CFTR Transmembrane-Domain Mutants by Mitigating their Interactions with Proteostasis
Miqueias Lopes-Pacheco et al.
CELLULAR PHYSIOLOGY AND BIOCHEMISTRY (2017)
Corrector VX-809 promotes interactions between cytoplasmic loop one and the first nucleotide-binding domain of CFTR
Tip W. Loo et al.
BIOCHEMICAL PHARMACOLOGY (2017)
Correctors Rescue CFTR Mutations in Nucleotide-Binding Domain 1 (NBD1) by Modulating Proteostasis
Miqueias Lopes-Pacheco et al.
CHEMBIOCHEM (2016)
Discovery of bioactive molecules from CuAAC click-chemistry-based combinatorial libraries
Xueshun Wang et al.
DRUG DISCOVERY TODAY (2016)
Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis
Johanna F. Dekkers et al.
SCIENCE TRANSLATIONAL MEDICINE (2016)
Monomerization and ER Relocalization of GRASP Is a Requisite for Unconventional Secretion of CFTR
Jiyoon Kim et al.
TRAFFIC (2016)
Combination of Correctors Rescue ΔF508-CFTR by Reducing Its Association with Hsp40 and Hsp27
Miqueias Lopes-Pacheco et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2015)
Protein Traffic Disorders: an Effective High-Throughput Fluorescence Microscopy Pipeline for Drug Discovery
Hugo M. Botelho et al.
SCIENTIFIC REPORTS (2015)
Rescue of NBD2 Mutants N1303K and S1235R of CFTR by Small-Molecule Correctors and Transcomplementation
Daniele Rapino et al.
PLOS ONE (2015)
Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function
Fredrick Van Goor et al.
JOURNAL OF CYSTIC FIBROSIS (2014)
Revertants, Low Temperature, and Correctors Reveal the Mechanism of F508del-CFTR Rescue by VX-809 and Suggest Multiple Agents for Full Correction
Carlos M. Farinha et al.
CHEMISTRY & BIOLOGY (2013)
VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1
Hong Yu Ren et al.
MOLECULAR BIOLOGY OF THE CELL (2013)
Correction of Both NBD1 Energetics and Domain Interface Is Required to Restore ΔF508 CFTR Folding and Function
Wael M. Rabeh et al.
CELL (2012)
Combining Global and Local Measures for Structure-Based Druggability Predictions
Andrea Volkamer et al.
JOURNAL OF CHEMICAL INFORMATION AND MODELING (2012)
Ouabain mimics low temperature rescue of F508del-CFTR in cystic fibrosis epithelial cells
Donglei Zhang et al.
FRONTIERS IN PHARMACOLOGY (2012)
Mechanisms of CFTR folding at the endoplasmic reticulum
Soo Jung Kim et al.
FRONTIERS IN PHARMACOLOGY (2012)
Rescue of the mutant CFTR chloride channel by pharmacological correctors and low temperature analyzed by gene expression profiling
Elvira Sondo et al.
AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY (2011)
Rescue of ΔF508-CFTR Trafficking via a GRASP-Dependent Unconventional Secretion Pathway
Heon Yung Gee et al.
CELL (2011)
Influence of cell background on pharmacological rescue of mutant CFTR
Nicoletta Pedemonte et al.
AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY (2010)
The Cystic Fibrosis-causing Mutation ΔF508 Affects Multiple Steps in Cystic Fibrosis Transmembrane Conductance Regulator Biogenesis
Patrick H. Thibodeau et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2010)
Analyzing the Topology of Active Sites: On the Prediction of Pockets and Subpockets
Andrea Volkamer et al.
JOURNAL OF CHEMICAL INFORMATION AND MODELING (2010)
Interplay between ER Exit Code and Domain Conformation in CFTR Misprocessing and Rescue
Gargi Roy et al.
MOLECULAR BIOLOGY OF THE CELL (2010)
Chemical and Biological Folding Contribute to Temperature-Sensitive ΔF508 CFTR Trafficking
Xiaodong Wang et al.
TRAFFIC (2008)
Revertant mutants G550E and 4RK rescue cystic fibrosis mutants in the first nucleotide-binding domain of CFTR by different mechanisms
Monica Roxo-Rosa et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)
Rescue of ΔF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules
F Van Goor et al.
AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY (2006)
MolDock: A new technique for high-accuracy molecular docking
Rene Thomsen et al.
JOURNAL OF MEDICINAL CHEMISTRY (2006)
Failure of cAMP agonists to activate rescued ΔF508 CFTR in CFBE41o- airway epithelial monolayers
Z Bebok et al.
JOURNAL OF PHYSIOLOGY-LONDON (2005)
Small-molecule correctors of defective ΔF508-CFTR cellular processing identified by high-throughput screening
N Pedemonte et al.
JOURNAL OF CLINICAL INVESTIGATION (2005)
Structure of nucleotide-binding domain 1 of the cystic fibrosis transmembrane conductance regulator
HA Lewis et al.
EMBO JOURNAL (2004)
COPII-dependent export of cystic fibrosis transmembrane conductance regulator from the ER uses a di-acidic exit code
XD Wang et al.
JOURNAL OF CELL BIOLOGY (2004)
Mutations in the nucleotide binding domain 1 signature motif region rescue processing and functional defects of cystic fibrosis transmembrane conductance regulator ΔF508
ACV deCarvalho et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2002)
Conformational and temperature-sensitive stability defects of the ΔF508 cystic fibrosis transmembrane conductance regulator in post-endoplasmic reticulum compartments
M Sharma et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2001)
The Protein Data Bank
HM Berman et al.
NUCLEIC ACIDS RESEARCH (2000)