ArticleBlood2008
Overnight transduction with foamyviral vectors restores the long-term repopulating activity of Fancc-/- stem cells.
Article in Blood, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
What it found
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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.
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.
Who cites it
19 citing papers in PubMed, 30 citations in OpenAlex.
- Recent Advances in the Molecular Design and Delivery Technology of mRNA for Vaccination Against Infectious Diseases.Frontiers in immunology · 2022Review
- Adenovirus vectors in hematopoietic stem cell genome editing.FEBS letters · 2019Review
- Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency.Molecular therapy. Methods & clinical development · 2016Article
- Pak2 regulates hematopoietic progenitor cell proliferation, survival, and differentiation.Stem cells (Dayton, Ohio) · 2015Article
- Gene therapy model of X-linked severe combined immunodeficiency using a modified foamy virus vector.PloS one · 2013Article
- A Pak1-PP2A-ERM signaling axis mediates F-actin rearrangement and degranulation in mast cells.Experimental hematology · 2013Article
- Large animal models for foamy virus vector gene therapy.Viruses · 2012Review
- Cell Membrane-associated heparan sulfate is a receptor for prototype foamy virus in human, monkey, and rodent cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Advances in foamy virus vector technology and disease correction could speed the path to clinical application.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Foamy virus vector-mediated gene correction of a mouse model of Wiskott-Aldrich syndrome.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Article
- Advances in Gene Delivery Systems.Pharmaceutical medicine · 2011Article
- Review
- Progress and prospects: foamy virus vectors enter a new age.Gene therapy · 2010Review
- Article
- Article
- Molecular biology of foamy viruses.Medical microbiology and immunology · 2010Review
- Foamy virus vectors for gene transfer.Expert opinion on biological therapy · 2009Review
- Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia.Mutation research · 2009Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 4 institutions in 3 countries.
Funding
Abstract
Fanconi anemia (FA) is a complex genetic disorder characterized by congenital abnormalities, bone marrow failure, and myeloid malignancies. Identification of 13 FA genes has been instrumental to explore gene transfer technologies aimed at correction of autologous FA-deficient stem cells. To date, 3 human FA stem cell gene therapy trials with standard 4-day transduction protocols using gammaretroviral vectors failed to provide clinical benefit. In addition, 2- to 4 day ex vivo manipulation of bone marrow from mice containing a disruption of the homologue of human FANCC (Fancc) results in a time-dependent increase in apoptosis and a risk for malignant transformation of hematopoietic cells. Here, we show that a 14-hour transduction period allows a foamyviral vector construct expressing the human FANCC cDNA to efficiently transduce murine FA stem cells with 1 to 2 proviral integrations per genome. Functionally, the repopulating activity of Fancc(-/-) stem cells from reconstituted mice expressing the recombinant FANCC transgene was comparable with wild-type controls. Collectively, these data provide evidence that short-term transduction of c-kit(+) cells with a foamyviral vector is sufficient for functional correction of a stem cell phenotype in a murine FA model. These data could have implications for future gene therapy trials for FA patients.
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Registered trials
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.