Published in Mol Ther on March 03, 2009
Gene Therapy for Wiskott-Aldrich Syndrome (TIGET-WAS) | NCT01515462
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Wiskott-Aldrich syndrome protein regulates lipid raft dynamics during immunological synapse formation. Immunity (2002) 1.69
WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function. Blood (2008) 1.65
WASP deficiency leads to global defects of directed leukocyte migration in vitro and in vivo. J Leukoc Biol (2005) 1.59
Wiskott-Aldrich syndrome protein deficiency leads to reduced B-cell adhesion, migration, and homing, and a delayed humoral immune response. Blood (2004) 1.56
Unregulated actin polymerization by WASp causes defects of mitosis and cytokinesis in X-linked neutropenia. J Exp Med (2007) 1.49
Defective Th1 cytokine gene transcription in CD4+ and CD8+ T cells from Wiskott-Aldrich syndrome patients. J Immunol (2006) 1.44
Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients. Gene Ther (2006) 1.29
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Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction. Blood (2006) 1.16
Impaired T-cell priming in vivo resulting from dysfunction of WASp-deficient dendritic cells. Blood (2007) 1.15
Cutting edge: selective requirement for the Wiskott-Aldrich syndrome protein in cytokine, but not chemokine, secretion by CD4+ T cells. J Immunol (2004) 1.10
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Improvement of migratory defects in a murine model of Wiskott-Aldrich syndrome gene therapy. Mol Ther (2008) 0.99
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Improved lentiviral vectors for Wiskott-Aldrich syndrome gene therapy mimic endogenous expression profiles throughout haematopoiesis. Gene Ther (2008) 0.85
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WASP and the phenotypic range associated with deficiency. Curr Opin Allergy Clin Immunol (2005) 0.81
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Unrelated bone marrow transplantation with a reduced toxicity myeloablative conditioning regimen in Wiskott-Aldrich syndrome. J Korean Med Sci (2008) 0.78
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An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol (2011) 6.44
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Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science (2013) 5.64
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol (2006) 5.58
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol (2007) 5.36
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science (2013) 5.34
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Loss of mismatched HLA in leukemia after stem-cell transplantation. N Engl J Med (2009) 3.38
Th17 cells express interleukin-10 receptor and are controlled by Foxp3⁻ and Foxp3+ regulatory CD4+ T cells in an interleukin-10-dependent manner. Immunity (2011) 3.38
The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J Clin Invest (2009) 3.34
Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med (2006) 3.22
Pax7-expressing satellite cells are indispensable for adult skeletal muscle regeneration. Development (2011) 3.18
Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nat Rev Genet (2009) 3.09
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Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters. Nat Biotechnol (2004) 2.78
Differentiation of Tr1 cells by immature dendritic cells requires IL-10 but not CD25+CD4+ Tr cells. Blood (2004) 2.76
Identification of proangiogenic TIE2-expressing monocytes (TEMs) in human peripheral blood and cancer. Blood (2007) 2.74
Forebrain ependymal cells are Notch-dependent and generate neuroblasts and astrocytes after stroke. Nat Neurosci (2009) 2.74
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J Clin Invest (2007) 2.63
Cleavage of the plasma membrane Na+/Ca2+ exchanger in excitotoxicity. Cell (2005) 2.63
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Defective regulatory and effector T cell functions in patients with FOXP3 mutations. J Clin Invest (2006) 2.58
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Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat Methods (2011) 2.30
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood (2007) 2.28
Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med (2009) 2.19
The receptor Ly108 functions as a SAP adaptor-dependent on-off switch for T cell help to B cells and NKT cell development. Immunity (2012) 2.19
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Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome. Blood (2009) 1.89
Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy. Blood (2009) 1.88
NK cell activation by dendritic cells (DCs) requires the formation of a synapse leading to IL-12 polarization in DCs. Blood (2004) 1.88
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Plasma cells require autophagy for sustainable immunoglobulin production. Nat Immunol (2013) 1.85
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