Transcomplementation by a Truncation Mutant of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Enhances ΔF508 Processing through a Biomolecular Interaction

Managing Cystic Fibrosis

(2011) Malena Cohen-Cymberknoh et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

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

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

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

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

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

Cl Transport in Complemented CF Bronchial Epithelial Cells Correlates with CFTR mRNA Expression Levels

(2008) Beate Illek et al.   CELLULAR PHYSIOLOGY AND BIOCHEMISTRY

Chaperone displacement from mutant cystic fibrosis transmembrane conductance regulator restores its function in human airway epithelia

(2008) Fei Sun et al.   FASEB JOURNAL

Cystic Fibrosis Transmembrane Regulator Missing the First Four Transmembrane Segments Increases Wild Type and ΔF508 Processing

(2008) Liudmila Cebotaru et al.   JOURNAL OF BIOLOGICAL CHEMISTRY