ArticleMolecular therapy : the journal of the American Society of Gene Therapy2012
Foamy virus vector-mediated gene correction of a mouse model of Wiskott-Aldrich syndrome.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 25 citations in OpenAlex.
- Review
- Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice.Molecular therapy. Nucleic acids · 2018Article
- Cellular Antisilencing Elements Support Transgene Expression from Herpes Simplex Virus Vectors in the Absence of Immediate Early Gene Expression.Journal of virology · 2018Article
- Rare Genetic Blood Disease Modeling in Zebrafish.Frontiers in genetics · 2018Review
- Clinical Manifestations and Pathophysiological Mechanisms of the Wiskott-Aldrich Syndrome.Journal of clinical immunology · 2018Review
- Foamy Virus Vector Carries a Strong Insulator in Its Long Terminal Repeat Which Reduces Its Genotoxic Potential.Journal of virology · 2018Article
- Characterization of a core region in the A2UCOE that confers effective anti-silencing activity.Scientific reports · 2017Article
- Article
- Article
- Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency.Molecular therapy. Methods & clinical development · 2016Article
- A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent and pluripotent stem cells.Nucleic acids research · 2015Article
- Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases.International journal of hematology · 2014Review
- High-titer foamy virus vector transduction and integration sites of human CD34(+) cell-derived SCID-repopulating cells.Molecular therapy. Methods & clinical development · 2014Article
- 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 · 2013Article
- Gene therapy model of X-linked severe combined immunodeficiency using a modified foamy virus vector.PloS one · 2013Article
- 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
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Authors and funding
5 authors at 4 institutions in 2 countries.
Funding
Abstract
The Wiskott-Aldrich syndrome (WAS) is an X-linked disorder characterized by eczema, thrombocytopenia and immunodeficiency. Hematopoietic cell transplantation can cure the disease and gene therapy is being tested as an alternative treatment option. In this study, we assessed the use of foamy virus (FV) vectors as a gene transfer system for WAS, using a Was knockout (KO) mouse model. Preliminary experiments using FV vectors expressing the green fluorescent protein under the transcriptional control of the endogenous WAS promoter or a ubiquitously acting chromatin opening element allowed us to define transduction conditions resulting in high (>40%) and long-term in-vivo marking of blood cells after transplantation. In following experiments, Was KO mice were treated with FV vectors containing the human WAS complementary DNA (cDNA). Transplanted animals expressed the WAS protein (WASp) in T and B lymphocytes, as well as platelets and showed restoration of both T-cell receptor-mediated responses and B-cell migration. We also observed recovery of platelet adhesion and podosome formation in dendritic cells (DCs) of treated mice. These data demonstrate that FV vectors can be effective for hematopoietic stem cell (HSC)-directed gene correction of WAS.
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