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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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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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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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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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Current progress on gene therapy for primary immunodeficiencies

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

2013

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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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Hypoxia-regulated transgene expression in experimental retinal and choroidal neovascularization

Gene therapy, 2003-06, Vol.10 (12), p.1049-1054

2003

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Gene therapy progress and prospects: gene therapy for severe combined immunodeficiency

Gene therapy, 2003-11, Vol.10 (24), p.1999-2004

2003

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Progress and Prospects: Gene Therapy Clinical Trials (Part 1)

Gene therapy, 2007-10, Vol.14 (20), p.1439-1447

2007

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T cell transduction and suicide with an enhanced mutant thymidine kinase

Gene therapy, 2002-06, Vol.9 (12), p.824-827

2002

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Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2 model of retinal degeneration

Gene therapy, 2003-03, Vol.10 (6), p.523-527

2003

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Efficient and Selective AAV2-Mediated Gene Transfer Directed to Human Vascular Endothelial Cells

Molecular therapy, 2001-09, Vol.4 (3), p.174-181

2001

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High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H

Human gene therapy, 1999-10, Vol.10 (15), p.2527-2537

1999

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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

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

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

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

Current progress on gene therapy for primary immunodeficiencies

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

Hypoxia-regulated transgene expression in experimental retinal and choroidal neovascularization

Gene therapy progress and prospects: gene therapy for severe combined immunodeficiency

Progress and Prospects: Gene Therapy Clinical Trials (Part 1)

T cell transduction and suicide with an enhanced mutant thymidine kinase

Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2 model of retinal degeneration

Efficient and Selective AAV2-Mediated Gene Transfer Directed to Human Vascular Endothelial Cells

Lentiviral gene therapy for X-linked chronic granulomatous disease

High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H

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