Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine

Long-term Culture and Cloning of Primary Human Bronchial Basal Cells that Maintain Multipotent Differentiation Capacity and CFTR Channel Function

(2018) Jennifer Ruth Peters-Hall et al.   AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY

Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR

(2018) Scott H. Donaldson et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Personalised CFTR pharmacotherapeutic response testing and therapy of cystic fibrosis

(2018) Cormac McCarthy et al.   EUROPEAN RESPIRATORY JOURNAL

Detection of CFTR function and modulation in primary human nasal cell spheroids

(2018) John J. Brewington et al.   Journal of Cystic Fibrosis

Comparison of ex vivo and in vitro intestinal cystic fibrosis models to measure CFTR-dependent ion channel activity

(2018) Domenique D. Zomer-van Ommen et al.   Journal of Cystic Fibrosis

Up-to-date and projected estimates of survival for people with cystic fibrosis using baseline characteristics: A longitudinal study using UK patient registry data

(2018) Ruth H. Keogh et al.   Journal of Cystic Fibrosis

Assessment of p.Phe508del-CFTR functional restoration in pediatric primary cystic fibrosis airway epithelial cells

(2018) Erika N. Sutanto et al.   PLoS One

Differentiated human airway organoids to assess infectivity of emerging influenza virus

(2018) Jie Zhou et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

(2018) John J. Brewington et al.   Jove-Journal of Visualized Experiments

A single-cell atlas of the airway epithelium reveals the CFTR-rich pulmonary ionocyte

(2018) Lindsey W. Plasschaert et al.   NATURE

A revised airway epithelial hierarchy includes CFTR-expressing ionocytes

(2018) Daniel T. Montoro et al.   NATURE

Induced pluripotent stem cells for treating cystic fibrosis: State of the science

(2018) Bette S. Pollard et al.   PEDIATRIC PULMONOLOGY

The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis

(2017) Maria Talamo Guevara et al.   Expert Opinion On Drug Safety

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

(2017) Steven M. Rowe et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

(2017) Jennifer L. Taylor-Cousar et al.   NEW ENGLAND JOURNAL OF MEDICINE

Conditionally reprogrammed primary airway epithelial cells maintain morphology, lineage and disease specific functional characteristics

(2017) Kelly M. Martinovich et al.   Scientific Reports

Take a deep breath and digest the material: organoids and biomaterials of the respiratory and digestive systems

(2017) Briana R. Dye et al.   MRS Communications

Airway Progenitor Clone Formation Is Enhanced by Y-27632–Dependent Changes in the Transcriptome

(2016) Susan D. Reynolds et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Dual SMAD Signaling Inhibition Enables Long-Term Expansion of Diverse Epithelial Basal Cells

(2016) Hongmei Mou et al.   Cell Stem Cell

Optimal correction of distinct CFTR folding mutants in rectal cystic fibrosis organoids

(2016) Johanna F. Dekkers et al.   EUROPEAN RESPIRATORY JOURNAL

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

(2016) Johanna F. Dekkers et al.   Science Translational Medicine

Animal Models of Cystic Fibrosis Pathology: Phenotypic Parallels and Divergences

(2016) Gillian M. Lavelle et al.   Biomed Research International

Classification of CFTR mutation classes

(2016) Fernando Augusto Lima Marson et al.   Lancet Respiratory Medicine

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

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

Notch2 Is Required for Inflammatory Cytokine-Driven Goblet Cell Metaplasia in the Lung

(2015) Henry Danahay et al.   Cell Reports

Cystic fibrosis

(2015) Felix Ratjen et al.   Nature Reviews Disease Primers

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

Ex vivo culture of the intestinal epithelium: strategies and applications

(2014) Marc Leushacke et al.   GUT

Cystic fibrosis: Toward personalized therapies

(2014) Pauline T. Ikpa et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

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

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

A functional CFTR assay using primary cystic fibrosis intestinal organoids

(2013) Johanna F Dekkers et al.   NATURE MEDICINE

Multicenter Intestinal Current Measurements in Rectal Biopsies from CF and Non-CF Subjects to Monitor CFTR Function

(2013) John P. Clancy et al.   PLoS One

Changes in transcriptome of native nasal epithelium expressing F508del-CFTR and intersecting data from comparable studies

(2013) Luka A Clarke et al.   RESPIRATORY RESEARCH

Development of primary human nasal epithelial cell cultures for the study of cystic fibrosis pathophysiology

(2012) F. de Courcey et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Evaluation of the intestinal current measurement method as a diagnostic test for cystic fibrosis

(2012) Malena Cohen-Cymberknoh et al.   PEDIATRIC PULMONOLOGY

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

Isolation and in vitro expansion of human colonic stem cells

(2011) Peter Jung et al.   NATURE 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

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

Human primary bronchial lung cell constructs: The new respiratory models

(2010) Kelly BéruBé et al.   TOXICOLOGY

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

(2009) Toshiro Sato et al.   NATURE

Physiological Measurements Confirming the Diagnosis of Cystic Fibrosis: the Sweat Test and Measurements of Transepithelial Potential Difference

(2009) C.J. Taylor et al.   Paediatric Respiratory Reviews

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