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Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome

Nature Medicine, 2022-01, Vol.28 (1), p.71-80

2022

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Progress and prospects: gene therapy for inherited immunodeficiencies

Gene therapy, 2009-11, Vol.16 (11), p.1285-1291

2009

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Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer

Leukemia, 2011-09, Vol.25 (9), p.1471-1483

2011

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Translational Mini‐Review Series on Immunodeficiency: Molecular defects in common variable immunodeficiency

Clinical and experimental immunology, 2007-09, Vol.149 (3), p.401-409

2007

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Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS

Gene therapy, 2009-05, Vol.16 (5), p.605-619

2009

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Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients

Gene therapy, 2007-03, Vol.14 (5), p.415-428

2007

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Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection

Gene therapy, 2012-03, Vol.19 (3), p.246-254

2012

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Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1

Gene therapy, 2002-03, Vol.9 (5), p.320-326

2002

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Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors

Gene therapy, 2009-04, Vol.16 (4), p.509-520

2009

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Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy

Gene therapy, 2005-04, Vol.12 (8), p.694-701

2005

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Neonatal dendritic cells are intrinsically biased against Th‐1 immune responses

Clinical and experimental immunology, 2002-04, Vol.128 (1), p.118-123

2002

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Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression

Gene therapy, 2008-08, Vol.15 (16), p.1167-1175

2008

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Ectopic retroviral expression of LMO2, but not IL2Rγ, blocks human T-cell development from CD34 + cells : implications for leukemogenesis in gene therapy

Leukemia, 2007-04, Vol.21 (4), p.754-763

2007

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Genetic Variants Associated With Neutrophil Function in Aggressive Periodontitis and Healthy Controls

Journal of periodontology (1970), 2010-04, Vol.81 (4), p.527-534

2010

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In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium

Gene therapy, 2001-11, Vol.8 (21), p.1665-1668

2001

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Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences

Gene therapy, 2005-04, Vol.12 (8), p.715-723

2005

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Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy

Nature genetics, 2000-07, Vol.25 (3), p.306-310

2000

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Chemotaxis of macrophages is abolished in the Wiskott‐Aldrich syndrome

British journal of haematology, 1998-06, Vol.101 (4), p.659-665

1998

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Rituximab for the treatment of autoimmune cytopenias in children with immune deficiency

British journal of haematology, 2007-07, Vol.138 (1), p.94-96

2007

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Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX

Gene therapy, 2006-01, Vol.13 (2), p.117-126

2006

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The Wiskott-Aldrich syndrome

Journal of Allergy and Clinical Immunology, 2006-04, Vol.117 (4), p.725-738

2006

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Wiskott-Aldrich syndrome protein is necessary for efficient IgG-mediated phagocytosis

Blood, 2000-05, Vol.95 (9), p.2943-2946

2000

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Immunotherapy for neuroblastoma using syngeneic fibroblasts transfected with IL-2 and IL-12

British journal of cancer, 2007-07, Vol.97 (2), p.210-217

2007

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Current progress on gene therapy for primary immunodeficiencies

Gene therapy, 2013-10, Vol.20 (10), p.963-969

2013

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Enhanced human cell engraftment in mice deficient in RAG2 and the common cytokine receptor γ chain

British journal of haematology, 1998-11, Vol.103 (2), p.335-342

1998

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Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome

Progress and prospects: gene therapy for inherited immunodeficiencies

Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer

Translational Mini‐Review Series on Immunodeficiency: Molecular defects in common variable immunodeficiency

Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS

Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients

Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection

Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1

Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors

Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy

Neonatal dendritic cells are intrinsically biased against Th‐1 immune responses

Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression

Ectopic retroviral expression of LMO2, but not IL2Rγ, blocks human T-cell development from CD34 + cells : implications for leukemogenesis in gene therapy

Genetic Variants Associated With Neutrophil Function in Aggressive Periodontitis and Healthy Controls

In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium

Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences

Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy

Chemotaxis of macrophages is abolished in the Wiskott‐Aldrich syndrome

Rituximab for the treatment of autoimmune cytopenias in children with immune deficiency

Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX

The Wiskott-Aldrich syndrome

Wiskott-Aldrich syndrome protein is necessary for efficient IgG-mediated phagocytosis

Immunotherapy for neuroblastoma using syngeneic fibroblasts transfected with IL-2 and IL-12

Current progress on gene therapy for primary immunodeficiencies

Enhanced human cell engraftment in mice deficient in RAG2 and the common cytokine receptor γ chain

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