Gene therapy of severe combined immunodeficiencies (Q55982397)

Nature Reviews Immunology

Europe PubMed Central

title

Gene therapy of severe combined immunodeficiencies (English)

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3

Marina Cavazzana-Calvo

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author name string

Alain Fischer

series ordinal

1

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Salima Hacein-Bey

series ordinal

2

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publication date

August 2002

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published in

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volume

2

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issue

8

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page(s)

615-621

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cites work

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T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

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

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

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Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

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Cloning of human adenosine deaminase cDNA and expression in mouse cells

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An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency

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T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

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

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Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease

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Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells

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Interleukin-2 receptor gamma chain mutation results in X-linked severe combined immunodeficiency in humans

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Defective IL7R expression in T(-)B(+)NK(+) severe combined immunodeficiency

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Cytokines and immunodeficiency diseases

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

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

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

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

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Identification of clonogenic common lymphoid progenitors in mouse bone marrow

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Identification of a novel, human multilymphoid progenitor in cord blood

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RAG mutations in human B cell-negative SCID.

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Artemis, a novel DNA double-strand break repair/V(D)J recombination protein, is mutated in human severe combined immune deficiency.

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HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia

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Preselective gene therapy for Fabry disease

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Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin

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Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

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Use of Serum-Free Medium with Fibronectin Fragment Enhanced Transduction in a System of Gas Permeable Plastic Containers to Achieve High Levels of Retrovirus Transduction at Clinical Scale

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Thymic function after hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency

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