Evidence map›Paper›PMID 21422098›Full record

ReviewHuman molecular genetics2011

Genetic therapies for cystic fibrosis lung disease.

Patrick L Sinn, Reshma M Anthony, Paul B McCray

Open access · bronzeAbstract readReview
In one paragraph

Review in Human molecular genetics, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed
4.6field-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

12 citing papers in PubMed, 34 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Article
  5. Article
  6. Lung gene therapy with highly compacted DNA nanoparticles that overcome the mucus barrier.Journal of controlled release : official journal of the Controlled Release Society · 2014
    Article
  7. Formulation of pH responsive peptides as inhalable dry powders for pulmonary delivery of nucleic acids.European journal of pharmaceutics and biopharmaceutics : official journal of Arbeitsgemeinschaft fur Pharmazeutische Verfahrenstechnik e.V · 2014
    Article
  8. Review
  9. Article
  10. Lentiviral vector gene transfer to porcine airways.Molecular therapy. Nucleic acids · 2012
    Article
  11. Transcriptional targeting in the airway using novel gene regulatory elements.American journal of respiratory cell and molecular biology · 2012
    Article
  12. Gene therapy for the treatment of cystic fibrosis.The application of clinical genetics · 2012
    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

3 authors at 1 institution in 1 country.

Patrick L SinnProgram in Gene Therapy, Department of Pediatrics, Carver College of Medicine, The University of Iowa, Iowa City, IA 52242, USA.
Reshma M Anthony
Paul B McCray
University of Iowa · US

Funding

Vector CoreP01HL051670 · NHLBI · UNIVERSITY OF IOWA · PI MCCRAY, PAUL B · 1993 to 2019
$36.5M
Vector Core-Core 2P30DK054759 · NIDDK · UNIVERSITY OF IOWA · PI Alejandro Antonio Pezzulo · 1998 to 2026
$30.5M
Filovirus enveloped FIV:virus-epithelia interactions-CFR01HL075363 · NHLBI · UNIVERSITY OF IOWA · PI MCCRAY, PAUL B · 2004 to 2007
$1.4M
Allele-Specific Repair of the CFTR DeltaF508 Mutation using Zinc Finger NucleasesR21HL091808 · NHLBI · UNIVERSITY OF IOWA · PI MCCRAY, PAUL B · 2008 to 2009
$460k
NHLBI NIH HHS P01 HL-51670NHLBI NIH HHS R01 HL-075363NHLBI NIH HHS R21 HL-91808NIDDK NIH HHS P30 DK054759NIDDK NIH HHS P30 DK-54759
6 · The paper itself

Abstract

The aim of gene therapy for cystic fibrosis (CF) lung disease is to efficiently and safely express the CF transmembrane conductance regulator (CFTR) in the appropriate pulmonary cell types. Although CF patients experience multi-organ disease, the chronic bacterial lung infections and associated inflammation are the primary cause of shortened life expectancy. Gene transfer-based therapeutic approaches are feasible, in part, because the airway epithelium is directly accessible by aerosol delivery or instillation. Improvements in standard delivery vectors and the development of novel vectors, as well as emerging technologies and new animal models, are propelling exciting new research forward. Here, we review recent developments that are advancing this field of investigation.

Indexed as

Cystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorEpitheliumGenetic TherapyGenetic VectorsHumansInflammationLungCystic Fibrosis Transmembrane Conductance Regulator

Identifiers

PMID21422098
PMCPMC3095059
OpenAlexW2161668762

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.