Evidence map›Paper›PMID 23727931›Full record

ArticleNature medicine2013

A functional CFTR assay using primary cystic fibrosis intestinal organoids.

Johanna F Dekkers, Caroline L Wiegerinck, Hugo R de Jonge, Inez Bronsveld, Hettie M Janssens, Karin M de Winter-de Groot, Arianne M Brandsma, Nienke W M de Jong, Marcel J C Bijvelds, Bob J Scholte and 6 more

2 registry-linked trialsAbstract read
PubMed Publisher
In one paragraph

Article in Nature medicine, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 541 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
541citing papers in PubMed, 1 pooled it
37.0field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02965326 narecruitingnot on this mapstarted 2016, after this paper: background citation

Personalized Therapy of Cystic Fibrosis: Set-up of Response Markers

TypeinterventionalSponsorHôpital Necker-Enfants MaladesRan2016 to 2026Enrolled75ConditionsCystic FibrosisArmsNasal swab, rectal biopsy.
NCT06322446 naactive not recruitingnot on this mapstarted 2024, after this paper: background citation

Effects of a Strength Exercise Program on Muscular Health in People With CF Treated With Modulators of the Cystic Fibrosis Transmembrane Conductance Regulator Receptor (CFTR)

TypeinterventionalSponsorUniversidad Politecnica de MadridRan2024 to 2026Enrolled48ConditionsCystic FibrosisArmsExercise
3 · Its place in the literature

Who cites it

541 citing papers in PubMed, 1 synthesis or guideline pooled it, 984 citations in OpenAlex.

  1. CRISPR for cystic fibrosis: Advances and insights from a systematic review.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
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  18. Organoids for Metabolic Disease Modeling.Journal of inherited metabolic disease · 2026
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481 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

16 authors at 4 institutions in 1 country.

Johanna F DekkersDepartment of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands.
Caroline L Wiegerinck
Hugo R de Jonge
Inez Bronsveld
Hettie M Janssens
Karin M de Winter-de Groot
Arianne M Brandsma
Nienke W M de Jong
Marcel J C Bijvelds
Bob J Scholte
Edward E S Nieuwenhuis
Stieneke van den Brink
Hans Clevers
Cornelis K van der Ent
Sabine Middendorp
Jeffrey M Beekman
University Medical Center Utrecht · NLWilhelmina Children's Hospital · NLErasmus MC · NLErasmus University Rotterdam · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

We recently established conditions allowing for long-term expansion of epithelial organoids from intestine, recapitulating essential features of the in vivo tissue architecture. Here we apply this technology to study primary intestinal organoids of people suffering from cystic fibrosis, a disease caused by mutations in CFTR, encoding cystic fibrosis transmembrane conductance regulator. Forskolin induces rapid swelling of organoids derived from healthy controls or wild-type mice, but this effect is strongly reduced in organoids of subjects with cystic fibrosis or in mice carrying the Cftr F508del mutation and is absent in Cftr-deficient organoids. This pattern is phenocopied by CFTR-specific inhibitors. Forskolin-induced swelling of in vitro-expanded human control and cystic fibrosis organoids corresponds quantitatively with forskolin-induced anion currents in freshly excised ex vivo rectal biopsies. Function of the CFTR F508del mutant protein is restored by incubation at low temperature, as well as by CFTR-restoring compounds. This relatively simple and robust assay will facilitate diagnosis, functional studies, drug development and personalized medicine approaches in cystic fibrosis.

Indexed as

AnimalsBiological AssayCells, CulturedCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorFemaleHEK293 CellsHumansIntestinal MucosaMaleMiceMice, Inbred C57BLMice, Inbred CFTRMutant ProteinsOrganoidsPrimary Cell CultureCystic Fibrosis Transmembrane Conductance RegulatorMutant ProteinsWater

Identifiers

PMID23727931
OpenAlexW2151829259

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.