Stem cell-derived organoids to model gastrointestinal facets of cystic fibrosis

rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice

(2016) Dragana Vidović et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Genome Editing in Human Pluripotent Stem Cells: Approaches, Pitfalls, and Solutions

(2016) William T. Hendriks et al.   Cell Stem Cell

Targeted CFTR gene disruption with zinc-finger nucleases in human intestinal epithelial cells induces oxidative stress and inflammation

(2016) Marie-Laure Kleme et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations

(2016) Gudio Veit et al.   MOLECULAR BIOLOGY OF THE CELL

Generating and regenerating the digestive system

(2016) James M. Wells   Nature Reviews Gastroenterology & Hepatology

Airway acidification initiates host defense abnormalities in cystic fibrosis mice

(2016) V. S. Shah et al.   SCIENCE

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

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

“Miniguts” from plucked human hair meet Crohn’s disease

(2016) M. Hohwieler et al.   ZEITSCHRIFT FUR GASTROENTEROLOGIE

Animal models of gastrointestinal and liver diseases. Animal models of cystic fibrosis: gastrointestinal, pancreatic, and hepatobiliary disease and pathophysiology

(2015) Alicia K. Olivier et al.   AMERICAN JOURNAL OF PHYSIOLOGY-GASTROINTESTINAL AND LIVER PHYSIOLOGY

Junctional abnormalities in human airway epithelial cells expressing F508del CFTR

(2015) Samuel A. Molina et al.   AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY

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

(2015) Meritxell Huch et al.   CELL

Prospective Derivation of a Living Organoid Biobank of Colorectal Cancer Patients

(2015) Marc van de Wetering et al.   CELL

Organoid Models of Human and Mouse Ductal Pancreatic Cancer

(2015) Sylvia F. Boj et al.   CELL

Loss of cftr function leads to pancreatic destruction in larval zebrafish

(2015) Adam Navis et al.   DEVELOPMENTAL BIOLOGY

Generating human intestinal tissues from pluripotent stem cells to study development and disease

(2015) K. L. Sinagoga et al.   EMBO JOURNAL

The cystic fibrosis transmembrane conductance regulator as a biomarker in non-small cell lung cancer

(2015) JIN LI et al.   INTERNATIONAL JOURNAL OF ONCOLOGY

Defective goblet cell exocytosis contributes to murine cystic fibrosis–associated intestinal disease

(2015) Jinghua Liu et al.   JOURNAL OF CLINICAL INVESTIGATION

CFTR Knockdown induces proinflammatory changes in intestinal epithelial cells

(2015) Karoline St-Martin Crites et al.   Journal of Inflammation-London

Cystic fibrosis mouse model-dependent intestinal structure and gut microbiome

(2015) Mark Bazett et al.   MAMMALIAN GENOME

Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation

(2015) Fotios Sampaziotis et al.   NATURE BIOTECHNOLOGY

A high-throughput platform for stem cell niche co-cultures and downstream gene expression analysis

(2015) Adam D. Gracz et al.   NATURE CELL BIOLOGY

Ductal pancreatic cancer modeling and drug screening using human pluripotent stem cell– and patient-derived tumor organoids

(2015) Ling Huang et al.   NATURE MEDICINE

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

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

High level of CFTR expression is associated with tumor aggression and knockdown of CFTR suppresses proliferation of ovarian cancer in vitro and in vivo

(2015) JIAO XU et al.   ONCOLOGY REPORTS

Mortality from cystic fibrosis in Europe: 1994-2010

(2015) Esther Quintana-Gallego et al.   PEDIATRIC PULMONOLOGY

Preserved genetic diversity in organoids cultured from biopsies of human colorectal cancer metastases

(2015) Fleur Weeber et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells

(2015) Ana M. Crane et al.   Stem Cell Reports

Human iPS Cell-Derived Insulin Producing Cells Form Vascularized Organoids under the Kidney Capsules of Diabetic Mice

(2015) Sudhanshu P. Raikwar et al.   PLoS One

Gastrointestinal Pathology in Juvenile and Adult CFTR-Knockout Ferrets

(2014) Xingshen Sun et al.   AMERICAN JOURNAL OF PATHOLOGY

Disrupted interaction between CFTR and AF-6/afadin aggravates malignant phenotypes of colon cancer

(2014) Ting Ting Sun et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Organoid cultures for the analysis of cancer phenotypes

(2014) Norman Sachs et al.   CURRENT OPINION IN GENETICS & DEVELOPMENT

Evidence for a Causal Relationship Between Early Exocrine Pancreatic Disease and Cystic Fibrosis–Related Diabetes: A Mendelian Randomization Study

(2014) David Soave et al.   DIABETES

Hepatic organoids for microfluidic drug screening

(2014) Sam H. Au et al.   LAB ON A CHIP

Characterization of cultured cholangiocytes isolated from livers of patients with primary sclerosing cholangitis

(2014) James H Tabibian et al.   LABORATORY INVESTIGATION

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

A Comparative View on Human Somatic Cell Sources for iPSC Generation

(2014) Stefanie Raab et al.   Stem Cells International

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

Human Intestinal Tissue with Adult Stem Cell Properties Derived from Pluripotent Stem Cells

(2014) Ryan Forster et al.   Stem Cell Reports

Update in Cystic Fibrosis 2012

(2013) Christopher H. Goss et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Lung Phenotype of Juvenile and Adult Cystic Fibrosis Transmembrane Conductance Regulator–Knockout Ferrets

(2013) Xingshen Sun et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Downregulation of CFTR promotes epithelial-to-mesenchymal transition and is associated with poor prognosis of breast cancer

(2013) Jie Ting Zhang et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

(2013) Gerald Schwank et al.   Cell Stem Cell

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

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

Unlimited in vitro expansion of adult bi-potent pancreas progenitors through the Lgr5/R-spondin axis

(2013) Meritxell Huch et al.   EMBO JOURNAL

Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs

(2013) David A. Stoltz et al.   JOURNAL OF CLINICAL INVESTIGATION

Exocrine cell-derived microparticles in response to lipopolysaccharide promote endocrine dysfunction in cystic fibrosis

(2013) Andrei Alexandru Constantinescu et al.   Journal of Cystic Fibrosis

In vitro expansion of single Lgr5+ liver stem cells induced by Wnt-driven regeneration

(2013) Meritxell Huch et al.   NATURE

Vascularized and functional human liver from an iPSC-derived organ bud transplant

(2013) Takanori Takebe et al.   NATURE

A network of epigenetic regulators guides developmental haematopoiesis in vivo

(2013) Hsuan-Ting Huang et al.   NATURE CELL BIOLOGY

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

(2013) Tsukasa Okiyoneda et al.   Nature Chemical Biology

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

The Cystic Fibrosis of Exocrine Pancreas

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

Pancreatic Damage in Fetal and Newborn Cystic Fibrosis Pigs Involves the Activation of Inflammatory and Remodeling Pathways

(2012) Maisam Abu-El-Haija et al.   AMERICAN JOURNAL OF PATHOLOGY

Pancreatic and biliary secretion are both altered in cystic fibrosis pigs

(2012) Aliye Uc et al.   AMERICAN JOURNAL OF PHYSIOLOGY-GASTROINTESTINAL AND LIVER PHYSIOLOGY

Generation of Multipotent Lung and Airway Progenitors from Mouse ESCs and Patient-Specific Cystic Fibrosis iPSCs

(2012) Hongmei Mou et al.   Cell Stem Cell

Intestinal pH and Gastrointestinal Transit Profiles in Cystic Fibrosis Patients Measured by Wireless Motility Capsule

(2012) Daniel Gelfond et al.   DIGESTIVE DISEASES AND SCIENCES

Abnormal endocrine pancreas function at birth in cystic fibrosis ferrets

(2012) Alicia K. Olivier et al.   JOURNAL OF CLINICAL INVESTIGATION

Ivacaftor potentiation of multiple CFTR channels with gating mutations

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

The challenges and promises of new therapies for cystic fibrosis: Figure 1.

(2012) Gerald B. Pier   JOURNAL OF EXPERIMENTAL MEDICINE

Generating cells of the gastrointestinal system: current approaches and applications for the differentiation of human pluripotent stem cells

(2012) Allen Wang et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Cancer Risk in Cystic Fibrosis: A 20-Year Nationwide Study From the United States

(2012) Patrick Maisonneuve et al.   JNCI-Journal of the National Cancer Institute

Directed differentiation of human pluripotent stem cells into mature airway epithelia expressing functional CFTR protein

(2012) Amy P Wong et al.   NATURE BIOTECHNOLOGY

Functional engraftment of colon epithelium expanded in vitro from a single adult Lgr5+ stem cell

(2012) Shiro Yui et al.   NATURE MEDICINE

CFTR suppresses tumor progression through miR-193b targeting urokinase plasminogen activator (uPA) in prostate cancer

(2012) C Xie et al.   ONCOGENE

Epithelial cell polarity: a major gatekeeper against cancer?

(2011) C Royer et al.   CELL DEATH AND DIFFERENTIATION

Genetics of pancreatitis

(2011) Jessica LaRusch et al.   CURRENT OPINION IN GASTROENTEROLOGY

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

(2011) Toshiro Sato et al.   GASTROENTEROLOGY

Disruption of cytokeratin-8 interaction with F508del-CFTR corrects its functional defect

(2011) Julien Colas et al.   HUMAN MOLECULAR GENETICS

Mouse models of cystic fibrosis: Phenotypic analysis and research applications

(2011) Martina Wilke et al.   Journal of Cystic Fibrosis

Sequential targeting of CFTR by BAC vectors generates a novel pig model of cystic fibrosis

(2011) N. Klymiuk et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Genome-wide association and linkage identify modifier loci of lung disease severity in cystic fibrosis at 11p13 and 20q13.2

(2011) Fred A Wright et al.   NATURE GENETICS

Isolation and in vitro expansion of human colonic stem cells

(2011) Peter Jung et al.   NATURE MEDICINE

Generating human intestinal tissue from pluripotent stem cells in vitro

(2011) Kyle W McCracken et al.   Nature Protocols

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

The K+ Channel Opener 1-EBIO Potentiates Residual Function of Mutant CFTR in Rectal Biopsies from Cystic Fibrosis Patients

(2011) Eva K. Roth et al.   PLoS One

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

Promoter hypermethylation of the CFTR gene and clinical/pathological features associated with non-small cell lung cancer

(2011) JI WOONG SON et al.   RESPIROLOGY

The  F508 Mutation Causes CFTR Misprocessing and Cystic Fibrosis-Like Disease in Pigs

(2011) L. S. Ostedgaard et al.   Science Translational Medicine

Pathology of Gastrointestinal Organs in a Porcine Model of Cystic Fibrosis

(2010) David K. Meyerholz et al.   AMERICAN JOURNAL OF PATHOLOGY

Pancreatic cancer in chronic pancreatitis; aetiology, incidence, and early detection

(2010) Sara Raimondi et al.   BEST PRACTICE & RESEARCH IN CLINICAL GASTROENTEROLOGY

Functional analysis of F508del CFTR in native human colon

(2010) Andrea van Barneveld et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Obesity adversely affects survival in pancreatic cancer patients

(2010) Robert R. McWilliams et al.   CANCER

Lgr5+ve Stem Cells Drive Self-Renewal in the Stomach and Build Long-Lived Gastric Units In Vitro

(2010) Nick Barker et al.   Cell Stem Cell

Dynamic Regulation of CFTR Bicarbonate Permeability by [Cl−]i and Its Role in Pancreatic Bicarbonate Secretion

(2010) Hyun Woo Park et al.   GASTROENTEROLOGY

Type of CFTR Mutation Determines Risk of Pancreatitis in Patients With Cystic Fibrosis

(2010) Chee Y. Ooi et al.   GASTROENTEROLOGY

Combined Bicarbonate Conductance-Impairing Variants in CFTR and SPINK1 Variants Are Associated With Chronic Pancreatitis in Patients Without Cystic Fibrosis

(2010) Alexander Schneider et al.   GASTROENTEROLOGY

Disease phenotype of a ferret CFTR-knockout model of cystic fibrosis

(2010) Xingshen Sun et al.   JOURNAL OF CLINICAL INVESTIGATION

Directed differentiation of human pluripotent stem cells into intestinal tissue in vitro

(2010) Jason R. Spence et al.   NATURE

Cystic Fibrosis Pigs Develop Lung Disease and Exhibit Defective Bacterial Eradication at Birth

(2010) D. A. Stoltz et al.   Science Translational Medicine

Generation of Transgene-Free Lung Disease-Specific Human Induced Pluripotent Stem Cells Using a Single Excisable Lentiviral Stem Cell Cassette

(2010) Aba Somers et al.   STEM CELLS

Molecular and cellular regulation of pancreatic duct cell function

(2009) Martin C Steward et al.   CURRENT OPINION IN GASTROENTEROLOGY

Genetic Modifiers of Liver Disease in Cystic Fibrosis

(2009) Jaclyn R. Bartlett   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

CFTR Functions as a Bicarbonate Channel in Pancreatic Duct Cells

(2009) Hiroshi Ishiguro et al.   JOURNAL OF GENERAL PHYSIOLOGY

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

(2009) Toshiro Sato et al.   NATURE

CFTR Is a Negative Regulator of NFκB Mediated Innate Immune Response

(2009) Neeraj Vij et al.   PLoS One

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

Adeno-associated virus–targeted disruption of the CFTR gene in cloned ferrets

(2008) Xingshen Sun et al.   JOURNAL OF CLINICAL INVESTIGATION

Disruption of the CFTR Gene Produces a Model of Cystic Fibrosis in Newborn Pigs

(2008) C. S. Rogers et al.   SCIENCE