Combination therapy with cystic fibrosis transmembrane conductance regulator modulators augment the airway functional microanatomy

Defective Innate Immunity and Hyperinflammation in Newborn Cystic Fibrosis Transmembrane Conductance Regulator–Knockout Ferret Lungs

(2015) Nicholas W. Keiser et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

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

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Breakthrough therapies: Cystic fibrosis (CF) potentiators and correctors

(2015) George M. Solomon et al.   PEDIATRIC PULMONOLOGY

Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis

(2014) Steven M. Rowe et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

A Functional Anatomic Defect of the Cystic Fibrosis Airway

(2014) Susan E. Birket et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

An Autoregulatory Mechanism Governing Mucociliary Transport Is Sensitive to Mucus Load

(2014) Linbo Liu et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiators Protect G551D but Not ΔF508 CFTR from Thermal Instability

(2014) Xuehong Liu et al.   BIOCHEMISTRY

Characterization of Defects in Ion Transport and Tissue Development in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Knockout Rats

(2014) Katherine L. Tuggle et al.   PLoS One

Impaired mucus detachment disrupts mucociliary transport in a piglet model of cystic fibrosis

(2014) M. J. Hoegger et al.   SCIENCE

Some gating potentiators, including VX-770, diminish  F508-CFTR functional expression

(2014) G. Veit et al.   Science Translational Medicine

Potentiator ivacaftor abrogates pharmacological correction of  F508 CFTR in cystic fibrosis

(2014) D. M. Cholon et al.   Science Translational Medicine

A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial

(2014) Michael P Boyle et al.   Lancet Respiratory Medicine

Native Small Airways Secrete Bicarbonate

(2013) A. K. M. Shamsuddin et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Change in Sweat Chloride as a Clinical End Point in Cystic Fibrosis Clinical Trials

(2013) Anthony G. Durmowicz et al.   CHEST

Sweat chloride as a biomarker of CFTR activity: Proof of concept and ivacaftor clinical trial data

(2013) Frank J. Accurso et al.   Journal of Cystic Fibrosis

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

(2013) Tsukasa Okiyoneda et al.   Nature Chemical Biology

Method for Quantitative Study of Airway Functional Microanatomy Using Micro-Optical Coherence Tomography

(2013) Linbo Liu et al.   PLoS One

Optimizing Nasal Potential Difference Analysis for CFTR Modulator Development: Assessment of Ivacaftor in CF Subjects with the G551D-CFTR Mutation

(2013) Steven M. Rowe et al.   PLoS One

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 Regulator Correctors and Potentiators

(2013) S. M. Rowe et al.   Cold Spring Harbor Perspectives in Medicine

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

A Dense Poly(Ethylene Glycol) Coating Improves Penetration of Large Polymeric Nanoparticles Within Brain Tissue

(2012) E. A. Nance et al.   Science Translational Medicine

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

Role of epithelial HCO3− transport in mucin secretion: lessons from cystic fibrosis

(2010) Paul M. Quinton   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

A new role for bicarbonate in mucus formation

(2010) Eric Y. T. Chen et al.   AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY

Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosis

(2010) Peter A Sloane et al.   CURRENT OPINION IN PULMONARY MEDICINE

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

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

ΔF508 CFTR processing correction and activity in polarized airway and non-airway cell monolayers

(2010) S.M. Rowe et al.   PULMONARY PHARMACOLOGY & THERAPEUTICS

Micro- and macrorheology of mucus

(2009) Samuel K. Lai et al.   ADVANCED DRUG DELIVERY REVIEWS

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

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

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

A2BAdenosine Receptors Regulate the Mucus Clearance Component of the Lung's Innate Defense System

(2008) Brett M. Rollins et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Clinical practice and genetic counseling for cystic fibrosis and CFTR-related disorders

(2008) Samuel M Moskowitz et al.   GENETICS IN MEDICINE

Cystic fibrosis: impaired bicarbonate secretion and mucoviscidosis

(2008) Paul M Quinton   LANCET