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

Nature Medicine, 2022-10, Vol.28 (10), p.2217-2217

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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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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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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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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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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Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency

Gene therapy, 2012-08, Vol.19 (8), p.872-876

2012

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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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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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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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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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Correction of SCID-X1 using an enhancerless Vav promoter

Human gene therapy, 2011-03, Vol.22 (3), p.263-270

2011

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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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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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Cutting Edge: The Wiskott-Aldrich Syndrome Protein Is Required for Efficient Phagocytosis of Apoptotic Cells

The Journal of immunology (1950), 2001-04, Vol.166 (8), p.4831-4834

2001

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Dendritic Cells: The Bare Bones of Immunity

Current biology, 2004-11, Vol.14 (22), p.R965-R967

2004

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Cell-specific and efficient expression in mouse and human B cells by a novel hybrid immunoglobulin promoter in a lentiviral vector

Gene therapy, 2007-12, Vol.14 (23), p.1623-1631

2007

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Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina

Gene therapy, 2003-08, Vol.10 (16), p.1336-1344

2003

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

Author Correction: 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

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

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

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

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

Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency

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

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

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

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

Correction of SCID-X1 using an enhancerless Vav promoter

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

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

Cutting Edge: The Wiskott-Aldrich Syndrome Protein Is Required for Efficient Phagocytosis of Apoptotic Cells

Dendritic Cells: The Bare Bones of Immunity

Cell-specific and efficient expression in mouse and human B cells by a novel hybrid immunoglobulin promoter in a lentiviral vector

Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina

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