Evidence map›Paper›PMID 18600482›Full record

ReviewClinical reviews in allergy & immunology2008

Gene therapy for cystic fibrosis.

Christian Mueller, Terence R Flotte

Abstract readReview
PubMed Publisher
In one paragraph

Review in Clinical reviews in allergy & immunology, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.

0numbers the graph read from it
0cells of the map it votes in
26citing papers in PubMed
–field-weighted citation impact
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

26 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. Article
  5. Review
  6. Restoration of F508-del Function by Transcomplementation: The Partners Meet in the Endoplasmic Reticulum.Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology · 2019
    Article
  7. Article
  8. Review
  9. Article
  10. Hybrid nonviral/viral vector systems for improved piggyBac DNA transposon in vivo delivery.Molecular therapy : the journal of the American Society of Gene Therapy · 2015
    Article
  11. Review
  12. Testing gene therapy vectors in human primary nasal epithelial cultures.Molecular therapy. Methods & clinical development · 2015
    Article
  13. Overcoming the cystic fibrosis sputum barrier to leading adeno-associated virus gene therapy vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2014
    Article
  14. Article
  15. Article
  16. Article
  17. Article
  18. Article
  19. Whole genome amplification in preimplantation genetic diagnosis.Journal of Zhejiang University. Science. B · 2011
    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.

Christian MuellerDepartment of Pediatrics, University of Massachusetts Medical School, Worcester, MA, USA.
Terence R Flotte

Funding

Vector CoreP01HL051811 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI GUGGINO, WILLIAM B. · 1993 to 2013
$20.2M
University of Massachusetts Center for Clinical and Translational ScienceUL1TR000161 · NCATS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI LUZURIAGA, KATHERINE F · 2012 to 2015
$11.0M
NCATS NIH HHS UL1 TR000161NHLBI NIH HHS P01 HL051811
6 · The paper itself

Abstract

Cystic Fibrosis (CF) is an autosomal recessive disorder due to mutations in the CF transmembrane conductance regulator (CFTR) gene that lead to defective ion transport in the conducting pulmonary airways and exocrine glands. Through a process that is not fully understood, CFTR defects predispose affected patients to chronic endobronchial infections with organisms such as Pseudomonas aeruginosa and Staphylococcus aureus. Following the discovery of the CFTR gene in 1989, CF became one of the primary targets for gene therapy research. Early enthusiasm surrounded the new field of gene therapy during most of the 1990s and it led academics and clinicians on a big effort to apply gene therapy for cystic fibrosis. Clinical studies have been pursued using recombinant adenovirus, recombinant adeno-associated virus, cationic liposomes, and cationic polymer vectors. Although to this date no dramatic therapeutic benefits have been observed, a lot of information has been gained from the pre-clinical and clinical studies that were performed. This learning curve has led to the optimization of vector technology and an appreciation of immune and mechanical barriers that have to be overcome for successful delivery.

Indexed as

Genetic TherapyAnimalsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorGenes, RecessiveGenetic Predisposition to DiseaseGenetic VectorsHumansImmunity, CellularMutagenesis, Site-DirectedProtein TransportSignal TransductionStem CellsTargeted Gene RepairCystic Fibrosis Transmembrane Conductance Regulator

Identifiers

What OpenQuestion holds

Textmetadata
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.