Binding screen for cystic fibrosis transmembrane conductance regulator correctors finds new chemical matter and yields insights into cystic fibrosis therapeutic strategy

Deletion of Phenylalanine 508 in the First Nucleotide-binding Domain of the Cystic Fibrosis Transmembrane Conductance Regulator Increases Conformational Exchange and Inhibits Dimerization

(2015) P. Andrew Chong et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Restoration of NBD1 Thermal Stability Is Necessary and Sufficient to Correct ∆F508 CFTR Folding and Assembly

(2015) Lihua He et al.   JOURNAL OF MOLECULAR BIOLOGY

VX-809 and Related Corrector Compounds Exhibit Secondary Activity Stabilizing Active F508del-CFTR after Its Partial Rescue to the Cell Surface

(2014) Paul D.W. Eckford et al.   CHEMISTRY & BIOLOGY

Discovery of novel potent ΔF508-CFTR correctors that target the nucleotide binding domain

(2013) Norbert Odolczyk et al.   EMBO Molecular Medicine

VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1

(2013) Hong Yu Ren et al.   MOLECULAR BIOLOGY OF THE CELL

Mechanism-based corrector combination restores ΔF508-CFTR folding and function

(2013) Tsukasa Okiyoneda et al.   Nature Chemical Biology

Vx-770 potentiates CFTR function by promoting decoupling between the gating cycle and ATP hydrolysis cycle

(2013) Kang-Yang Jih et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Cystic Fibrosis Transmembrane Conductance Regulator (ABCC7) Structure

(2013) J. F. Hunt et al.   Cold Spring Harbor Perspectives in Medicine

Dynamics Intrinsic to Cystic Fibrosis Transmembrane Conductance Regulator Function and Stability

(2013) P. A. Chong et al.   Cold Spring Harbor Perspectives in Medicine

Fragment-Based Approaches in Drug Discovery and Chemical Biology

(2012) Duncan E. Scott et al.   BIOCHEMISTRY

Requirements for Efficient Correction of ΔF508 CFTR Revealed by Analyses of Evolved Sequences

(2012) Juan L. Mendoza et al.   CELL

Correction of Both NBD1 Energetics and Domain Interface Is Required to Restore ΔF508 CFTR Folding and Function

(2012) Wael M. Rabeh et al.   CELL

Fragment Based Drug Design: From Experimental to Computational Approaches

(2012) A. Kumar et al.   CURRENT MEDICINAL CHEMISTRY

Conformational Changes Relevant to Channel Activity and Folding within the first Nucleotide Binding Domain of the Cystic Fibrosis Transmembrane Conductance Regulator

(2012) Rhea P. Hudson et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiator VX-770 (Ivacaftor) Opens the Defective Channel Gate of Mutant CFTR in a Phosphorylation-dependent but ATP-independent Manner

(2012) Paul D. W. Eckford et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

The Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)

(2011) Mark F. Rosenberg et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Design of a multi-purpose fragment screening library using molecular complexity and orthogonal diversity metrics

(2011) Wan F. Lau et al.   JOURNAL OF COMPUTER-AIDED MOLECULAR DESIGN

Fragment Based Drug Discovery: Practical Implementation Based on 19F NMR Spectroscopy

(2011) John B. Jordan et al.   JOURNAL OF MEDICINAL CHEMISTRY

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

(2011) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for theF508del-CFTRmutation

(2011) J P Clancy et al.   THORAX

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

Structures of a minimal human CFTR first nucleotide-binding domain as a monomer, head-to-tail homodimer, and pathogenic mutant

(2010) Shane Atwell et al.   PROTEIN ENGINEERING DESIGN & SELECTION

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

NMR evidence for differential phosphorylation-dependent interactions in WT and ΔF508 CFTR

(2009) Voula Kanelis et al.   EMBO 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

The rise of fragment-based drug discovery

(2009) Christopher W. Murray et al.   Nature Chemistry

CFTR Function and Prospects for Therapy

(2008) John R. Riordan   Annual Review of Biochemistry

Solubilizing Mutations Used to Crystallize One CFTR Domain Attenuate the Trafficking and Channel Defects Caused by the Major Cystic Fibrosis Mutation

(2008) Luísa S. Pissarra et al.   CHEMISTRY & BIOLOGY