Probing Conformational Rescue Induced by a Chemical Corrector of F508del-Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutant

Identification of a NBD1-Binding Pharmacological Chaperone that Corrects the Trafficking Defect of F508del-CFTR

(2011) Heidi M. Sampson et al.   CHEMISTRY & BIOLOGY

Restoration of domain folding and interdomain assembly by second-site suppressors of the ΔF508 mutation in CFTR

(2010) Lihua He et al.   FASEB JOURNAL

The Cystic Fibrosis-causing Mutation ΔF508 Affects Multiple Steps in Cystic Fibrosis Transmembrane Conductance Regulator Biogenesis

(2010) Patrick H. Thibodeau et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Regulatory Insertion Removal Restores Maturation, Stability and Function of ΔF508 CFTR

(2010) Andrei A. Aleksandrov et al.   JOURNAL OF MOLECULAR BIOLOGY

A Chemical Corrector Modifies the Channel Function of F508del-CFTR

(2010) P. K. Chiaw et al.   MOLECULAR PHARMACOLOGY

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation

(2010) Frank J. Accurso et al.   NEW ENGLAND JOURNAL OF MEDICINE

The Primary Folding Defect and Rescue of ΔF508 CFTR Emerge during Translation of the Mutant Domain

(2010) Hanneke Hoelen et al.   PLoS One

Thermal unfolding studies show the disease causing F508del mutation in CFTR thermodynamically destabilizes nucleotide-binding domain 1

(2010) Irina Protasevich et al.   PROTEIN SCIENCE

Integrated biophysical studies implicate partial unfolding of NBD1 of CFTR in the molecular pathogenesis of F508del cystic fibrosis

(2010) Chi Wang et al.   PROTEIN SCIENCE

Molecular models of the open and closed states of the whole human CFTR protein

(2009) Jean-Paul Mornon et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Functional Rescue of DeltaF508-CFTR by Peptides Designed to Mimic Sorting Motifs

(2009) Patrick Kim Chiaw et al.   CHEMISTRY & BIOLOGY

A truncated CFTR protein rescues endogenous ΔF508-CFTR and corrects chloride transport in mice

(2009) Estelle Cormet-Boyaka et al.   FASEB JOURNAL

Structure and Dynamics of NBD1 from CFTR Characterized Using Crystallography and Hydrogen/Deuterium Exchange Mass Spectrometry

(2009) H.A. Lewis et al.   JOURNAL OF MOLECULAR BIOLOGY

Interplay between ER Exit Code and Domain Conformation in CFTR Misprocessing and Rescue

(2009) Gargi Roy et al.   MOLECULAR BIOLOGY OF THE CELL

A Small-Molecule Modulator Interacts Directly with  Phe508-CFTR to Modify Its ATPase Activity and Conformational Stability

(2009) L. Wellhauser et al.   MOLECULAR PHARMACOLOGY

Strict coupling between CFTR's catalytic cycle and gating of its Cl- ion pore revealed by distributions of open channel burst durations

(2009) L. Csanady et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

(2009) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Structure of P-Glycoprotein Reveals a Molecular Basis for Poly-Specific Drug Binding

(2009) S. G. Aller et al.   SCIENCE

Correctors promote folding of the CFTR in the endoplasmic reticulum

(2008) Tip W. Loo et al.   BIOCHEMICAL JOURNAL

Atomic model of human cystic fibrosis transmembrane conductance regulator: Membrane-spanning domains and coupling interfaces

(2008) J.-P. Mornon et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Multiple Membrane-Cytoplasmic Domain Contacts in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mediate Regulation of Channel Gating

(2008) Lihua He et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Phenylalanine-508 mediates a cytoplasmic-membrane domain contact in the CFTR 3D structure crucial to assembly and channel function

(2008) A. W. R. Serohijos et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA