ArticleRare diseases (Austin, Tex.)2013
Novel opportunities for CFTR-targeting drug development using organoids.
Article in Rare diseases (Austin, Tex.), 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.
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.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
35 citing papers in PubMed, 93 citations in OpenAlex.
- Review
- Culture-specific transcriptional drifts limit the fidelity of organoid infection models.PLoS pathogens · 2026Article
- Towards functional precision medicine? Evidence standards of organoids as patient-specific models.History and philosophy of the life sciences · 2026Article
- Biomedical applications of organoids derived from the digestive system.Frontiers in cell and developmental biology · 2025Review
- Biobanks as an Indispensable Tool in the "Era" of Precision Medicine: Key Role in the Management of Complex Diseases, Such as Melanoma.Journal of personalized medicine · 2024Review
- The Effect of Complex Alleles of theInternational journal of molecular sciences · 2023Review
- Study Transportation of Drugs within Newly Established Murine Colon Organoid Systems.Advanced biology · 2023Article
- Article
- Clinical and Functional Characteristics of the E92KInternational journal of molecular sciences · 2023Article
- Evaluation of the Complex p.[Leu467Phe;Phe508del] CFTR Allele in the Intestinal Organoids Model: Implications for Therapy.International journal of molecular sciences · 2022Article
- Molecular Dynamics and Theratyping in Airway and Gut Organoids Reveal R352Q-CFTR Conductance Defect.American journal of respiratory cell and molecular biology · 2022Article
- Human organoids in basic research and clinical applications.Signal transduction and targeted therapy · 2022Review
- Review
- Culture and Imaging of Human Nasal Epithelial Organoids.Journal of visualized experiments : JoVE · 2021Article
- Organoids to model the endometrium: implantation and beyond.Reproduction & fertility · 2021Review
- Article
- Current Trends and Research Topics Regarding Intestinal Organoids: An Overview Based on Bibliometrics.Frontiers in cell and developmental biology · 2021Review
- New Therapeutic Approaches in Cystic Fibrosis.Turkish journal of pharmaceutical sciences · 2020Article
- Accumulation and persistence of ivacaftor in airway epithelia with prolonged treatment.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2020Article
- Biobanks-A Platform for Scientific and Biomedical Research.Diagnostics (Basel, Switzerland) · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR mutations lead to production of non-functional CFTR, reduced amounts of normal functioning CFTR or misfolded CFTR with defects in trafficking or function. For decades, CF treatment has been focused on the symptoms of CF, but pharmacotherapy using small molecules that target the basic defect of CF, the mutant CFTR protein, is now possible for a limited amount of subjects with CF. This raises the exciting possibility that the majority of people with CF may receive effective treatment targeting the different CFTR mutants in the future. We recently described a functional CFTR assay using rectal biopsies from subjects with CF that were cultured in vitro into self-organizing mini-guts or organoids. We here describe how this model may assist in the discovery of new CFTR-targeting drugs, the subjects that may benefit from these drugs, and the mechanisms underlying variability in CFTR genotype-phenotype relations.
Indexed as
Identifiers
What OpenQuestion holds
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.