Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases (Q34401676)

1 August 2017

title

Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases (English)

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1 August 2017

1

Fabio Candotti

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1 August 2017

publication date

1 February 2014

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International Journal of Hematology

1 August 2017

published in

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1 August 2017

volume

99

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1 August 2017

issue

4

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1 August 2017

page(s)

383-392

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Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

1 August 2017

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Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency

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Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

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Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

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Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

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Foamy virus vector-mediated gene correction of a mouse model of Wiskott-Aldrich syndrome

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How I treat ADA deficiency

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Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients

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Gene-transferred oligoclonal T cells predominantly persist in peripheral blood from an adenosine deaminase-deficient patient during gene therapy

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Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

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Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial.

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Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency

21 January 2018

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Chronic granulomatous disease: overview and hematopoietic stem cell transplantation

21 January 2018

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Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

21 January 2018

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Gene therapy for primary immunodeficiencies: Part 2.

21 January 2018

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Restoration of NET formation by gene therapy in CGD controls aspergillosis

21 January 2018

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Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial

21 January 2018

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Gene therapy of chronic granulomatous disease: the engraftment dilemma

21 January 2018

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Simian immunodeficiency virus lentivector corrects human X-linked chronic granulomatous disease in the NOD/SCID mouse xenograft

21 January 2018

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Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells

21 January 2018

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Lymphoid development and function in X-linked severe combined immunodeficiency mice after stem cell gene therapy

21 January 2018

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Lack of dominant-negative effects of a truncated gamma(c) on retroviral-mediated gene correction of immunodeficient mice.

21 January 2018

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Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model.

21 January 2018

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Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy

21 January 2018

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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

21 January 2018

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HTLV-1 Tax oncoprotein stimulates ROS production and apoptosis in T cells by interacting with USP10.

21 January 2018

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A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

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Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

21 January 2018

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Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice

21 January 2018

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Defects in T-cell-mediated immunity to influenza virus in murine Wiskott-Aldrich syndrome are corrected by oncoretroviral vector-mediated gene transfer into repopulating hematopoietic cells

21 January 2018

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A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice.

21 January 2018

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21 January 2018

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Improvement of migratory defects in a murine model of Wiskott-Aldrich syndrome gene therapy

21 January 2018

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Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models

21 January 2018

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Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome

21 January 2018

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Somatic mosaicism in Wiskott--Aldrich syndrome suggests in vivo reversion by a DNA slippage mechanism.

21 January 2018

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Second-site mutation in the Wiskott-Aldrich syndrome (WAS) protein gene causes somatic mosaicism in two WAS siblings

21 January 2018

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Multiple patients with revertant mosaicism in a single Wiskott-Aldrich syndrome family.

21 January 2018

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https://api.crossref.org/works/10.1007%2FS12185-014-1524-Z

Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction.

21 January 2018

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https://api.crossref.org/works/10.1007%2FS12185-014-1524-Z

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

21 January 2018

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Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting

21 January 2018

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https://api.crossref.org/works/10.1007%2FS12185-014-1524-Z

Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells.

21 January 2018

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Complete correction of murine Artemis immunodeficiency by lentiviral vector-mediated gene transfer

21 January 2018

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Construction of retroviral vectors carrying human CD3 gamma cDNA and reconstitution of CD3 gamma expression and T cell receptor surface expression and function in a CD3 gamma-deficient mutant T cell line

21 January 2018

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In vitro correction of JAK3-deficient severe combined immunodeficiency by retroviral-mediated gene transduction

21 January 2018

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Signaling via IL-2 and IL-4 in JAK3-deficient severe combined immunodeficiency lymphocytes: JAK3-dependent and independent pathways.

21 January 2018

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Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer

21 January 2018

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Virus-specific immunity after gene therapy in a murine model of severe combined immunodeficiency

21 January 2018

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Self-selection by genetically modified committed lymphocyte precursors reverses the phenotype of JAK3-deficient mice without myeloablation

21 January 2018

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https://api.crossref.org/works/10.1007%2FS12185-014-1524-Z

Correction of CD18-deficient lymphocytes by retrovirus-mediated gene transfer

21 January 2018

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Retroviral mediated expression of CD18 in normal and deficient human bone marrow progenitor cells

21 January 2018

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Correction of the disease phenotype in canine leukocyte adhesion deficiency using ex vivo hematopoietic stem cell gene therapy.

21 January 2018

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Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors

21 January 2018

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Lentiviral vectors incorporating a human elongation factor 1alpha promoter for the treatment of canine leukocyte adhesion deficiency

21 January 2018

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Gene therapy of canine leukocyte adhesion deficiency using lentiviral vectors with human CD11b and CD18 promoters driving canine CD18 expression.

21 January 2018

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Gene therapy for canine leukocyte adhesion deficiency with lentiviral vectors using the murine stem cell virus and human phosphoglycerate kinase promoters

21 January 2018

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Lentivirus gene therapy for purine nucleoside phosphorylase deficiency

21 January 2018

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Long-term immune reconstitution in RAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity

21 January 2018

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Restoration of human B-cell differentiation into NOD-SCID mice engrafted with gene-corrected CD34+ cells isolated from Artemis or RAG1-deficient patients

21 January 2018

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

21 January 2018

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Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency

21 January 2018

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Human adenylate kinase 2 deficiency causes a profound hematopoietic defect associated with sensorineural deafness

21 January 2018

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Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice

21 January 2018

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Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency.

21 January 2018

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A tissue-specific, activation-inducible, lentiviral vector regulated by human CD40L proximal promoter sequences

21 January 2018

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Sustained correction of B-cell development and function in a murine model of X-linked agammaglobulinemia (XLA) using retroviral-mediated gene transfer

21 January 2018

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Development of an enhanced B-specific lentiviral vector expressing BTK: a tool for gene therapy of XLA.

21 January 2018

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B cell-specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia

21 January 2018

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Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized human BTK.

21 January 2018

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SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease

21 January 2018

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Reconstitution of T cell receptor signaling in ZAP-70-deficient cells by retroviral transduction of the ZAP-70 gene.

21 January 2018

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Retrovirus-mediated transduction of primary ZAP-70-deficient human T cells results in the selective growth advantage of gene-corrected cells: implications for gene therapy

21 January 2018

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Reconstitution of lymphoid development and function in ZAP-70-deficient mice following gene transfer into bone marrow cells

21 January 2018

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In vivo correction of ZAP-70 immunodeficiency by intrathymic gene transfer

21 January 2018

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ZAP-70 restoration in mice by in vivo thymic electroporation

21 January 2018

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Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99

21 January 2018

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Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation

21 January 2018

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Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency

21 January 2018

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12 December 2020

Retroviral-mediated gene transfer of the leukocyte integrin CD18 into peripheral blood CD34+ cells derived from a patient with leukocyte adhesion deficiency type 1.

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12 December 2020

Gene therapy for chronic granulomatous disease

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12 December 2020

Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency

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12 December 2020

Correction of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantation

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12 December 2020

Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome

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12 December 2020

Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor

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12 December 2020

Gene therapy for leukocyte adhesion deficiency.

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12 December 2020

Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction

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12 December 2020

Improved transfer of the leukocyte integrin CD18 subunit into hematopoietic cell lines by using retroviral vectors having a gibbon ape leukemia virus envelope.

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12 December 2020

Restoration of lymphoid populations in a murine model of X-linked severe combined immunodeficiency by a gene-therapy approach

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12 December 2020

Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program

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12 December 2020

Correction of proliferative responses in purine nucleoside phosphorylase (PNP)-deficient T lymphocytes by retroviral-mediated PNP gene transfer and expression

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12 December 2020

Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability

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12 December 2020

Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia

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12 December 2020

Bone marrow gene transfer in three patients with adenosine deaminase deficiency.

1 reference

12 December 2020

Correction of defective expression in MHC class II deficiency (bare lymphocyte syndrome) cells by retroviral transduction of CIITA

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12 December 2020

Stable and functional lymphoid reconstitution of common cytokine receptor gamma chain deficient mice by retroviral-mediated gene transfer

1 reference

12 December 2020

10.1007/S12185-014-1524-Z

Identifiers

DOI

1 reference

Dimensions Publication ID

1 August 2017

1046367014

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