Evidence map›Paper›PMID 29731717›Full record

ReviewFrontiers in pharmacology2018

Innovative Therapeutic Strategies for Cystic Fibrosis: Moving Forward to CRISPR Technique.

Michele Marangi, Giuseppa Pistritto

Open access · goldAbstract readReview
In one paragraph

Review in Frontiers in pharmacology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed
4.2field-weighted citation impact, top 5% 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

24 citing papers in PubMed, 55 citations in OpenAlex.

  1. Review
  2. Next generation triplex-forming PNAs for site-specific genome editing of the F508del CFTR mutation.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2025
    Article
  3. Advances in Cystic Fibrosis Research in Qatar: A Commentary.Journal of personalized medicine · 2023
    Article
  4. Article
  5. Therapeutic Applications of the CRISPR-Cas System.Bioengineering (Basel, Switzerland) · 2022
    Review
  6. Review
  7. Review
  8. Article
  9. Article
  10. Article
  11. Review
  12. CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of Human Diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Review
  13. Review
  14. Enhancing Cystic Fibrosis Immune Regulation.Frontiers in pharmacology · 2021
    Article
  15. Review
  16. New Therapeutic Approaches in Cystic Fibrosis.Turkish journal of pharmaceutical sciences · 2020
    Article
  17. Review
  18. Novel therapeutic approaches for the management of cystic fibrosis.Multidisciplinary respiratory medicine · 2020
    Article
  19. Review
  20. 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

2 authors at 2 institutions in 1 country.

Michele MarangiDepartment of Economic Strategy of Pharmaceutical Products, Italian Medicines Agency, Rome, Italy.
Giuseppa PistrittoDepartment of Economic Strategy of Pharmaceutical Products, Italian Medicines Agency, Rome, Italy.
Italian Medicines Agency · ITUniversity of Rome Tor Vergata · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

One of the most revolutionary technologies in recent years in the field of molecular biology is CRISPR-Cas9. CRISPR technology is a promising tool for gene editing that provides researchers the opportunity to easily alter DNA sequences and modify gene function. Its many potential applications include correcting genetic defects, treating and preventing the spread of diseases. Cystic fibrosis (CF) is one of the most common lethal genetic diseases caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Although CF is an old acquaintance, there is still no effective/resolutive cure. Life expectancy has improved thanks to the combination of various treatments, but it is generally below average. Recently, a significant number of additional key medications have become licensed in Europe for the CF treatment including CFTR modulators. But innovative genomically-guided therapies have begun for CF and it is predictable that this will lead to rapid improvements in CF clinical disease and survival in the next decades. In this way, CRISPR-Cas9 approach may represent a valid tool to repair the CFTR mutation and hopeful results were obtained in tissue and animal models of CF disease.

Indexed as

CRISPR gene editingCTFR mutationcystic fibrosisgene therapyivacaftorlumacaftor

Identifiers

PMID29731717
PMCPMC5920621
OpenAlexW2797669280

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.