Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

Human Enteroids as a Model of Upper Small Intestinal Ion Transport Physiology and Pathophysiology

(2016) Jennifer Foulke-Abel et al.   GASTROENTEROLOGY

Mechanosensitive activation of CFTR by increased cell volume and hydrostatic pressure but not shear stress

(2015) Constanze Vitzthum et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

Long-Term Culture of Genome-Stable Bipotent Stem Cells from Adult Human Liver

(2015) Meritxell Huch et al.   CELL

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

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

New pharmacological approaches for cystic fibrosis: Promises, progress, pitfalls

(2015) Scott C. Bell et al.   PHARMACOLOGY & THERAPEUTICS

Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation

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

Cystic fibrosis genetics: from molecular understanding to clinical application

(2014) Garry R. Cutting   NATURE REVIEWS GENETICS

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

Adult Stem Cells in the Small Intestine Are Intrinsically Programmed with Their Location-Specific Function

(2014) Sabine Middendorp et al.   STEM CELLS

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

Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function

(2013) Fredrick Van Goor et al.   Journal of Cystic Fibrosis

Development of a primary mouse intestinal epithelial cell monolayer culture system to evaluate factors that modulate IgA transcytosis

(2013) C Moon et al.   Mucosal Immunology

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

(2013) Tsukasa Okiyoneda et al.   Nature Chemical Biology

Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene

(2013) Patrick R Sosnay et al.   NATURE GENETICS

A functional CFTR assay using primary cystic fibrosis intestinal organoids

(2013) Johanna F Dekkers et al.   NATURE MEDICINE

Growing Self-Organizing Mini-Guts from a Single Intestinal Stem Cell: Mechanism and Applications

(2013) T. Sato et al.   SCIENCE

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

(2012) Patrick A. Flume et al.   CHEST

Repeatability and bronchodilator reversibility of lung function in young children

(2012) Samatha Sonnappa et al.   EUROPEAN RESPIRATORY JOURNAL

Utilização da técnica de resistência do interruptor na avaliação da resistência das vias aéreas em pacientes com fibrose cística

(2012) Alessandra Rocha et al.   Jornal Brasileiro de Pneumologia

Ivacaftor potentiation of multiple CFTR channels with gating mutations

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

Peyer's Patch M Cells Derived from Lgr5+ Stem Cells Require SpiB and Are Induced by RankL in Cultured "Miniguts"

(2012) W. de Lau et al.   MOLECULAR AND CELLULAR BIOLOGY

Measurements of CFTR-Mediated Cl− Secretion in Human Rectal Biopsies Constitute a Robust Biomarker for Cystic Fibrosis Diagnosis and Prognosis

(2012) Marisa Sousa et al.   PLoS One

Long-term Expansion of Epithelial Organoids From Human Colon, Adenoma, Adenocarcinoma, and Barrett's Epithelium

(2011) Toshiro Sato et al.   GASTROENTEROLOGY

New clinical diagnostic procedures for cystic fibrosis in Europe

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

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

The n-of-1 clinical trial: the ultimate strategy for individualizing medicine?

(2011) Elizabeth O Lillie et al.   Personalized Medicine

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

Intestinal current measurement for diagnostic classification of patients with questionable cystic fibrosis: validation and reference data

(2010) N. Derichs et al.   THORAX

Single Lgr5 stem cells build crypt-villus structures in vitro without a mesenchymal niche

(2009) Toshiro Sato et al.   NATURE