Managing the Underlying Cause of Cystic Fibrosis: A Future Role for Potentiators and Correctors

Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with aG551DMutation

(2013) Jane C. Davies et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Pitfalls of Drug Development: Lessons Learned from Trials of Denufosol in Cystic Fibrosis

(2013) Richard B. Moss   JOURNAL OF PEDIATRICS

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

Correction of F508del-CFTR Trafficking by the Sponge Alkaloid Latonduine Is Modulated by Interaction with PARP

(2012) Graeme W. Carlile et al.   CHEMISTRY & BIOLOGY

Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

(2012) Patrick A. Flume et al.   CHEST

Ivacaftor potentiation of multiple CFTR channels with gating mutations

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

Long term effects of denufosol tetrasodium in patients with cystic fibrosis

(2012) Felix Ratjen et al.   Journal of Cystic Fibrosis

Bicarbonate and functional CFTR channel are required for proper mucin secretion and link cystic fibrosis with its mucus phenotype

(2012) Jenny K. Gustafsson et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Association of TMEM16A chloride channel overexpression with airway goblet cell metaplasia

(2012) Paolo Scudieri et al.   JOURNAL OF PHYSIOLOGY-LONDON

Advances in Cell and Gene-based Therapies for Cystic Fibrosis Lung Disease

(2012) Mayumi Oakland et al.   MOLECULAR THERAPY

Reduced airway surface pH impairs bacterial killing in the porcine cystic fibrosis lung

(2012) Alejandro A. Pezzulo et al.   NATURE

Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis

(2011) M. Wilschanski et al.   EUROPEAN RESPIRATORY JOURNAL

New clinical diagnostic procedures for cystic fibrosis in Europe

(2011) K. De Boeck et al.   Journal of Cystic Fibrosis

Suppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54

(2011) Steven M. Rowe et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

(2011) Bonnie W. Ramsey et al.   NEW ENGLAND JOURNAL OF 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

Influence of cell background on pharmacological rescue of mutant CFTR

(2010) Nicoletta Pedemonte et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis

(2010) Isabelle Sermet-Gaudelus et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Cystic fibrosis and survival to 40 years: a case-control study

(2010) N. J. Simmonds et al.   EUROPEAN RESPIRATORY JOURNAL

TMEM16A Inhibitors Reveal TMEM16A as a Minor Component of Calcium-activated Chloride Channel Conductance in Airway and Intestinal Epithelial Cells

(2010) Wan Namkung et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

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

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

Peripheral Protein Quality Control Removes Unfolded CFTR from the Plasma Membrane

(2010) T. Okiyoneda et al.   SCIENCE

Normal mouse intestinal mucus release requires cystic fibrosis transmembrane regulator–dependent bicarbonate secretion

(2009) Mary Abigail S. Garcia et al.   JOURNAL OF CLINICAL INVESTIGATION

Mutation-Specific Potency and Efficacy of Cystic Fibrosis Transmembrane Conductance Regulator Chloride Channel Potentiators

(2009) A. Caputo et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

Gating of the CFTR Cl−channel by ATP-driven nucleotide-binding domain dimerisation

(2009) Tzyh-Chang Hwang et al.   JOURNAL OF PHYSIOLOGY-LONDON

Mechanisms for Rescue of Correctable Folding Defects in CFTRΔF508

(2009) Diane E. Grove et al.   MOLECULAR BIOLOGY OF THE CELL

Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis

(2009) Darren M Hutt et al.   Nature Chemical Biology

Chloride channels as drug targets

(2009) Alan S. Verkman et al.   NATURE REVIEWS DRUG DISCOVERY

SPLUNC1 regulates airway surface liquid volume by protecting ENaC from proteolytic cleavage

(2009) A. Garcia-Caballero 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

CFTR Function and Prospects for Therapy

(2008) John R. Riordan   Annual Review of Biochemistry

Expression Cloning of TMEM16A as a Calcium-Activated Chloride Channel Subunit

(2008) Björn Christian Schroeder et al.   CELL

Cystic Fibrosis, Vector-Mediated Gene Therapy, and Relevance of Toll-Like Receptors: A Review of Problems, Progress, and Possibilities

(2008) Timothy Atkinson   CURRENT GENE THERAPY

An international/multicentre report on patients with cystic fibrosis (CF) over the age of 40 years

(2008) Margaret E. Hodson et al.   Journal of Cystic Fibrosis

Pharmacological Properties of N-(3,5-Diamino-6-chloropyrazine-2-carbonyl)-N'-4-[4-(2,3-dihydroxypropoxy)phenyl]butyl-guanidine Methanesulfonate (552-02), a Novel Epithelial Sodium Channel Blocker with Potential Clinical Efficacy for Cystic Fibrosis Lung Disease

(2008) A. J. Hirsh et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

TMEM16A confers receptor-activated calcium-dependent chloride conductance

(2008) Young Duk Yang et al.   NATURE

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

TMEM16A, A Membrane Protein Associated with Calcium-Dependent Chloride Channel Activity

(2008) A. Caputo et al.   SCIENCE