A novel treatment of cystic fibrosis acting on-target: cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR

Translating the genetics of cystic fibrosis to personalized medicine

(2016) Harriet Corvol et al.   Translational Research

Sweating the small stuff: Adequacy and accuracy in sweat chloride determination

(2015) Mari L. DeMarco et al.   CLINICAL BIOCHEMISTRY

Searching for a cure for cystic fibrosis. A 25-year quest in a nutshell

(2015) Barbara Bosch et al.   EUROPEAN JOURNAL OF PEDIATRICS

Inflammation in cystic fibrosis lung disease: Pathogenesis and therapy

(2015) André M. Cantin et al.   Journal of Cystic Fibrosis

Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients

(2015) M. D. Amaral   JOURNAL OF INTERNAL MEDICINE

Personalized medicine: Time for one-person trials

(2015) Nicholas J. Schork   NATURE

Another Beginning for Cystic Fibrosis Therapy

(2015) Pamela B. Davis   NEW ENGLAND JOURNAL OF MEDICINE

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

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

Precision Medicine — Personalized, Problematic, and Promising

(2015) J. Larry Jameson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Lumacaftor/ivacaftor for patients homozygous for Phe508del-CFTR: should we curb our enthusiasm?

(2015) A. M. Jones et al.   THORAX

Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis

(2015) Nikhil T. Awatade et al.   EBioMedicine

Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation

(2014) Daniela De Stefano et al.   Autophagy

The relative frequency of CFTR mutation classes in European patients with cystic fibrosis

(2014) K. De Boeck et al.   Journal of Cystic Fibrosis

Tissue Transglutaminase Activation Modulates Inflammation in Cystic Fibrosis via PPAR  Down-Regulation

(2014) L. Maiuri et al.   JOURNAL OF IMMUNOLOGY

Nephropathic cystinosis: an international consensus document

(2014) F. Emma et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

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

Disease-relevant proteostasis regulation of cystic fibrosis transmembrane conductance regulator

(2013) V R Villella et al.   CELL DEATH AND DIFFERENTIATION

Rescuing Mutant CFTR: A Multi-task Approach to a Better Outcome in Treating Cystic Fibrosis

(2013) Margarida D. Amaral et al.   CURRENT PHARMACEUTICAL DESIGN

Altered Expression and Localization of Ion Transporters Contribute to Diarrhea in Mice With Salmonella-Induced Enteritis

(2013) Ronald R. Marchelletta et al.   GASTROENTEROLOGY

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

Detection of Phospho-Sites Generated by Protein Kinase CK2 in CFTR: Mechanistic Aspects of Thr1471 Phosphorylation

(2013) Andrea Venerando et al.   PLoS One

Targeting autophagy as a novel strategy for facilitating the therapeutic action of potentiators on ΔF508 cystic fibrosis transmembrane conductance regulator

(2012) Alessandro Luciani et al.   Autophagy

CFTR biomarkers: time for promotion to surrogate end-point

(2012) K. De Boeck et al.   EUROPEAN RESPIRATORY JOURNAL

Understanding the natural progression in %FEV1decline in patients with cystic fibrosis: a longitudinal study

(2012) David Taylor-Robinson et al.   THORAX

Methods for stable recording of short-circuit current in a Na+-transporting epithelium

(2011) Veronika Gondzik et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

A randomized trial of recombinant human granulocyte-macrophage colony stimulating factor for patients with acute lung injury*

(2011) Robert Paine et al.   CRITICAL CARE MEDICINE

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

Defective CFTR induces aggresome formation and lung inflammation in cystic fibrosis through ROS-mediated autophagy inhibition

(2010) Alessandro Luciani et al.   NATURE CELL BIOLOGY

Chloride channels as drug targets

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