Evidence map›Paper›PMID 34740114›Full record

ReviewEBioMedicine2021

Perspectives on the translation of in-vitro studies to precision medicine in Cystic Fibrosis.

Marie-Pier Dumas, Sunny Xia, Christine E Bear, Felix Ratjen

Open access · goldAbstract readReview
In one paragraph

Review in EBioMedicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
2.1field-weighted citation impact, top 12% 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

11 citing papers in PubMed, 21 citations in OpenAlex.

  1. Review
  2. Review
  3. Human-based complexFrontiers in cell and developmental biology · 2025
    Review
  4. Article
  5. Cystic fibrosis.Nature reviews. Disease primers · 2024
    Review
  6. Article
  7. Review
  8. Who Modifies the Modifiers: A High-Resolution View of the Genetic Modifiers of Cystic Fibrosis.American journal of respiratory and critical care medicine · 2023
    Article
  9. Article
  10. Review
  11. Article
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

4 authors at 2 institutions in 1 country.

Marie-Pier DumasRespiratory Medicine, Hospital for Sick Children, Toronto, Canada; Translational Medicine, Hospital for Sick Children, Toronto, Canada.
Sunny XiaMolecular Medicine, Hospital for Sick Children, Toronto, Canada.; Department of Physiology, University of Toronto, Toronto, Canada.
Christine E BearMolecular Medicine, Hospital for Sick Children, Toronto, Canada.; Department of Physiology, University of Toronto, Toronto, Canada; Department of Biochemistry University of Toronto, Toronto, Canada.
Felix RatjenRespiratory Medicine, Hospital for Sick Children, Toronto, Canada; Translational Medicine, Hospital for Sick Children, Toronto, Canada.
Hospital for Sick Children · CAUniversity of Toronto · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Recent strides towards precision medicine in Cystic Fibrosis (CF) have been made possible by patient-derived in-vitro assays with the potential to predict clinical response to small molecule-based therapies. Here, we discuss the status of primary and stem-cell derived tissues used to evaluate the preclinical efficacy of CFTR modulators highlighting both their potential and limitations. Validation of these assays requires correlation of in-vitro responses to in-vivo measures of clinical biomarkers of disease outcomes. While initial efforts have shown some success, this translation requires methodologies that are sensitive enough to capture treatment responses in a CF population that now predominantly has mild lung disease. Future development of in-vitro and in-vivo biomarkers will facilitate the generation of new therapeutics particularly for those patients with rare mutations where clinical trials are not feasible so that in the future every CF patient will have access to effective targeted therapies.

Indexed as

Precision MedicineTranslational Research, BiomedicalAnimalsCell- and Tissue-Based TherapyCell Culture TechniquesCombined Modality TherapyCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDisease ManagementDisease SusceptibilityDrug DiscoveryGene EditingGenetic Predisposition to DiseaseHumansInduced Pluripotent Stem CellsOrganoidsCystic Fibrosis Transmembrane Conductance RegulatorCFTR modulatorsCystic FibrosisIn-vitro modelsOrganoidsOutcome measuresPrimary tissue

Identifiers

PMID34740114
PMCPMC8577330
OpenAlexW3208167843

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.