A functional CFTR assay using primary cystic fibrosis intestinal organoids (Q28291898)

1

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Caroline L Wiegerinck

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Hugo R de Jonge

3

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Inez Bronsveld

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Hettie M Janssens

5

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Karin M de Winter-de Groot

6

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Arianne M Brandsma

7

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Nienke W M de Jong

8

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Marcel J C Bijvelds

9

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Bob J Scholte

10

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Edward E S Nieuwenhuis

11

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Stieneke van den Brink

12

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Cornelis K van der Ent

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Sabine Middendorp

15

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Jeffrey M Beekman

Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA

16

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language of work or name

English

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publication date

July 2013

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published in

Nature Medicine

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volume

19

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issue

7

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page(s)

939-45

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cites work

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Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

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

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Rescue of ΔF508-CFTR trafficking via a GRASP-dependent unconventional secretion pathway

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CFTR inhibitors for treating diarrheal disease

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Lgr5 homologues associate with Wnt receptors and mediate R-spondin signalling

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Constitutive transcriptional activation by a beta-catenin-Tcf complex in APC-/- colon carcinoma

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Paneth cells constitute the niche for Lgr5 stem cells in intestinal crypts

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Identification of the cystic fibrosis gene: genetic analysis

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Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis

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CFTR function and prospects for therapy

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A CFTR potentiator in patients with cystic fibrosis and the G551D mutation

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Correction of both NBD1 energetics and domain interface is required to restore ΔF508 CFTR folding and function

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PTC124 targets genetic disorders caused by nonsense mutations

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Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis

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Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation.

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Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation

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Intestinal ion transport and the pathophysiology of diarrhea

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Ion transport in the small intestine

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Chloride secretion in a morphologically differentiated human colonic cell line that expresses the epithelial Na+ channel

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Discovery of glycine hydrazide pore-occluding CFTR inhibitors: mechanism, structure-activity analysis, and in vivo efficacy

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Production of a severe cystic fibrosis mutation in mice by gene targeting

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A delta F508 mutation in mouse cystic fibrosis transmembrane conductance regulator results in a temperature-sensitive processing defect in vivo

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Mouse models of cystic fibrosis: phenotypic analysis and research applications

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Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive

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Rescue of DeltaF508 and other misprocessed CFTR mutants by a novel quinazoline compound

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Small-molecule correctors of defective DeltaF508-CFTR cellular processing identified by high-throughput screening.

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Identification of mutations in regions corresponding to the two putative nucleotide (ATP)-binding folds of the cystic fibrosis gene

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Genotype and phenotype in cystic fibrosis

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Ex vivo CF diagnosis by intestinal current measurements (ICM) in small aperture, circulating Ussing chambers

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New clinical diagnostic procedures for cystic fibrosis in Europe

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Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809.

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Functional Cftr in crypt epithelium of organotypic enteroid cultures from murine small intestine

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Trafficking of immature DeltaF508-CFTR to the plasma membrane and its detection by biotinylation

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Coupling of cell volume and membrane potential changes to fluid secretion in a model of renal cysts

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Fluid and electrolyte transport by cultured human airway epithelia

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The K+ channel opener 1-EBIO potentiates residual function of mutant CFTR in rectal biopsies from cystic fibrosis patients

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

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Syntenin-mediated regulation of Sox4 proteasomal degradation modulates transcriptional output

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Impact of the cystic fibrosis mutation F508del-CFTR on renal cyst formation and growth

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A small molecule CFTR inhibitor produces cystic fibrosis-like submucosal gland fluid secretions in normal airways

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12 December 2020

Spectrum of mutations in the CFTR gene of patients with classical and atypical forms of cystic fibrosis from southwestern Sweden: identification of 12 novel mutations

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Personalized medicine in cystic fibrosis: dawning of a new era

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

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Identification of the L927P and delta L1260 mutations in the CFTR gene

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Association between genotype and pulmonary phenotype in cystic fibrosis patients with severe mutations

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cAMP-stimulated ion currents in Xenopus oocytes expressing CFTR cRNA

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12 December 2020

Identifiers

DOI

10.1038/NM.3201

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Consolidated OpenCitations Corpus – April 2017

release_7dykp253abb4hksttayeemcski

1808707

Fatcat ID

0 references

1808707

1 reference

Consolidated OpenCitations Corpus – April 2017

Consolidated OpenCitations Corpus – April 2017

1 reference