Capturing the Direct Binding of CFTR Correctors to CFTR by Using Click Chemistry

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

(2015) Claire E. Wainwright et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Young Hispanic with c.1646G>A Mutation Exhibits Severe Cystic Fibrosis Lung Disease: Is Ivacaftor an Option for Therapy?

(2013) Sunitha Yarlagadda et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE 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

Compartmentalization of cyclic nucleotide signaling: a question of when, where, and why?

(2013) Kavisha Arora et al.   PFLUGERS ARCHIV-EUROPEAN JOURNAL OF PHYSIOLOGY

Cystic Fibrosis Transmembrane Conductance Regulator–Modifying Medications: The Future of Cystic Fibrosis Treatment

(2012) Rebecca S Pettit   ANNALS OF PHARMACOTHERAPY

Click-based synthesis of triazolobithiazole ΔF508-CFTR correctors for cystic fibrosis

(2012) Michael B. Donald et al.   BIOORGANIC & MEDICINAL CHEMISTRY

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

(2012) Juan L. Mendoza et al.   CELL

Correctors of ΔF508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein

(2012) Lihua He et al.   FASEB JOURNAL

Ivacaftor potentiation of multiple CFTR channels with gating mutations

(2012) Haihui Yu et al.   Journal of Cystic Fibrosis

Promising new era dawns for cystic fibrosis treatment

(2012) Zöe Corbyn   LANCET

Increasing the Efficacy of Bioorthogonal Click Reactions for Bioconjugation: A Comparative Study

(2011) Christen Besanceney-Webler et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Rescue of ΔF508-CFTR Trafficking via a GRASP-Dependent Unconventional Secretion Pathway

(2011) Heon Yung Gee et al.   CELL

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 Transmembrane Conductance Regulator Impedes Proteolytic Stimulation of the Epithelial Na+Channel

(2010) Martina Gentzsch et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Peripheral Protein Quality Control Removes Unfolded CFTR from the Plasma Membrane

(2010) T. Okiyoneda et al.   SCIENCE

Functional Stability of Rescued ΔF508 Cystic Fibrosis Transmembrane Conductance Regulator in Airway Epithelial Cells

(2009) Asta Jurkuvenaite et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Correctors Enhance Maturation of ΔF508 CFTR by Promoting Interactions between the Two Halves of the Molecule

(2009) Tip W. Loo et al.   BIOCHEMISTRY

Click Chemistry and Bioorthogonal Reactions: Unprecedented Selectivity in the Labeling of Biological Molecules

(2009) Michael D. Best   BIOCHEMISTRY

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

Front-runners for pharmacotherapeutic correction of the airway ion transport defect in cystic fibrosis

(2008) M CLUNES et al.   CURRENT OPINION IN PHARMACOLOGY