Foamy Virus Vector-mediated Gene Correction of a Mouse Model of Wiskott–Aldrich Syndrome

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 (F...

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Veröffentlicht in:	Molecular therapy 2012-06, Vol.20 (6), p.1270-1279
Hauptverfasser:	Uchiyama, Toru, Adriani, Marsilio, Jagadeesh, G Jayashree, Paine, Adam, Candotti, Fabio
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals B-Lymphocytes - metabolism Blood Platelets - metabolism Cell Line Cells Cytomegalovirus Dendritic Cells - metabolism Disease Models, Animal Efficiency Female Foamy virus Gene Expression Gene Order Gene therapy Gene Transfer Techniques Genetic Therapy Genetic Vectors - administration & dosage Genomes Hematopoietic Stem Cells - metabolism Humans Hymenoptera Infections Male Mice Mice, Knockout Molecular biology Original Physiology Proteins Signal transduction Spumavirus - genetics T-Lymphocytes - metabolism Thrombocytopenia Transduction, Genetic Transgenes Vectors (Biology) Virus Integration Wiskott-Aldrich Syndrome - genetics Wiskott-Aldrich Syndrome - therapy Wiskott-Aldrich Syndrome Protein - genetics Wiskott-Aldrich Syndrome Protein - metabolism
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