Evidence map›Paper›PMID 20164857›Full record

ArticleGene therapy2010

Lentiviral vectors incorporating a human elongation factor 1alpha promoter for the treatment of canine leukocyte adhesion deficiency.

E J R Nelson, L M Tuschong, M J Hunter, T R Bauer, T H Burkholder, D D Hickstein

Open access · bronzeAbstract read
In one paragraph

Article in Gene therapy, 2010. 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
2.3field-weighted citation impact, top 11% 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, 26 citations in OpenAlex.

  1. Article
  2. Article
  3. Review
  4. Gene therapy in India: a focus.Journal of biosciences · 2014
    Review
  5. Review
  6. Long-term follow-up of foamy viral vector-mediated gene therapy for canine leukocyte adhesion deficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2013
    Article
  7. Review
  8. The function of dog models in developing gene therapy strategies for human health.Mammalian genome : official journal of the International Mammalian Genome Society · 2011
    Review
  9. Article
  10. Article
  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

6 authors at 3 institutions in 1 country.

E J R NelsonExperimental Transplantation and Immunology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892-1203, USA.
L M Tuschong
M J Hunter
T R Bauer
T H Burkholder
D D Hickstein
Center for Cancer Research · USNational Institutes of Health · USOffice of Research Services · US

Funding

Hematopoietic Stem Cell Gene Therapy for Leukocyte Adhesion DeficiencyZIASC010384 · NCI · DIVISION OF CLINICAL SCIENCES - NCI · PI HICKSTEIN, DENNIS · 2009 to 2025
$7.2M
Intramural NIH HHS ZIA SC010384
6 · The paper itself

Abstract

Canine leukocyte adhesion deficiency (CLAD) provides a unique large animal model for testing new therapeutic approaches for the treatment of children with leukocyte adhesion deficiency (LAD). In our CLAD model, we examined two different fragments of the human elongation factor 1alpha (EF1alpha) promoter (EF1alphaL, 1189 bp and EF1alphaS, 233 bp) driving the expression of canine CD18 in a self-inactivating (SIN) lentiviral vector. The EF1alphaS vector resulted in the highest levels of canine CD18 expression in CLAD CD34(+) cells in vitro. Subsequently, autologous CD34(+) bone marrow cells from four CLAD pups were transduced with the EF1alphaS vector and infused following a non-myeloablative dose of 200 cGy total-body irradiation. None of the CLAD pups achieved levels of circulating CD18(+) neutrophils sufficient to reverse the CLAD phenotype, and all four animals were euthanized because of infections within 9 weeks of treatment. These results indicate that the EF1alphaS promoter-driven CD18 expression in the context of a RRLSIN lentiviral vector does not lead to sufficient numbers of CD18(+) neutrophils in vivo to reverse the CLAD phenotype when used in a non-myeloablative transplant regimen in dogs.

Indexed as

Genetic VectorsLentivirusPromoter Regions, GeneticAnimalsAntigens, CD34Bone MarrowBone Marrow TransplantationCD18 AntigensDisease Models, AnimalDogsGenetic TherapyHumansLeukocyte-Adhesion Deficiency SyndromeNeutrophilsPeptide Elongation Factor 1Transduction, GeneticAntigens, CD34CD18 AntigensPeptide Elongation Factor 1

Identifiers

PMID20164857
PMCPMC3461956
OpenAlexW2052936135

What OpenQuestion holds

Textmetadata
LicenceTDM
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.