Gene therapy for primary immunodeficiencies. (Q38364511)

2 September 2017

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title

Gene therapy for primary immunodeficiencies. (English)

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2 September 2017

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1

A Fischer

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Marina Cavazzana

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4

object named as

M Cavazzana

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Salima Hacein-Bey-Abina

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2

object named as

S Hacein-Bey Abina

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Fabien Touzot

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F Touzot

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3

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publication date

24 February 2015

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2 September 2017

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2 September 2017

88

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2 September 2017

507-515

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2 September 2017

6

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Gene therapy of severe combined immunodeficiencies.

2 September 2017

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Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?

21 January 2018

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Transplantation outcomes for severe combined immunodeficiency, 2000-2009.

21 January 2018

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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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HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders

21 January 2018

1 reference

Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study

21 January 2018

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20 years of gene therapy for SCID.

21 January 2018

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Duration of antiviral immunity after smallpox vaccination

21 January 2018

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Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus

21 January 2018

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Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse

21 January 2018

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Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production

21 January 2018

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

21 January 2018

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

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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Efficacy of gene therapy for X-linked severe combined immunodeficiency

21 January 2018

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

21 January 2018

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High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR).

21 January 2018

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DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

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

21 January 2018

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Transcription start regions in the human genome are favored targets for MLV integration

21 January 2018

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

21 January 2018

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Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency.

21 January 2018

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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.

21 January 2018

1 reference

How I treat severe combined immunodeficiency

21 January 2018

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

21 January 2018

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Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

21 January 2018

1 reference

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.

21 January 2018

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Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

21 January 2018

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Cell competition is a tumour suppressor mechanism in the thymus.

21 January 2018

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Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity

21 January 2018

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WASP: a key immunological multitasker

21 January 2018

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Stem-cell gene therapy for the Wiskott-Aldrich syndrome

21 January 2018

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Chronic granulomatous disease. Report on a national registry of 368 patients

21 January 2018

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Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

21 January 2018

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

21 January 2018

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In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

21 January 2018

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Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.

21 January 2018

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Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors

21 January 2018

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Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

21 January 2018

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Assessing the risk of T-cell malignancies in mouse models of SCID-X1.

21 January 2018

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A modified γ-retrovirus vector for X-linked severe combined immunodeficiency

21 January 2018

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Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

21 January 2018

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

21 January 2018

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Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome

21 January 2018

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Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome

21 January 2018

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Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

21 January 2018

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Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

21 January 2018

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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

21 January 2018

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Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy

21 January 2018

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Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome

21 January 2018

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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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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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Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.

21 January 2018

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Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter.

21 January 2018

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Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

21 January 2018

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Leukocyte adhesion deficiencies

21 January 2018

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Long-term follow-up of foamy viral vector-mediated gene therapy for canine leukocyte adhesion deficiency.

21 January 2018

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Molecular mechanisms of biogenesis and exocytosis of cytotoxic granules

21 January 2018

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X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease

21 January 2018

1 reference

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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21 January 2018

FOXP3: of mice and men.

1 reference

Treatment of the Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome (IPEX) by Allogeneic Bone Marrow Transplantation

21 January 2018

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CD4⁺ T cells from IPEX patients convert into functional and stable regulatory T cells by FOXP3 gene transfer

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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Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model

21 January 2018

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Transduction of human CD34+ repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line.

21 January 2018

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Baboon envelope pseudotyped LVs outperform VSV-G-LVs for gene transfer into early-cytokine-stimulated and resting HSCs.

21 January 2018

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Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells

21 January 2018

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Prostaglandin E2 enhances human cord blood stem cell xenotransplants and shows long-term safety in preclinical nonhuman primate transplant models.

21 January 2018

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Aryl hydrocarbon receptor antagonists promote the expansion of human hematopoietic stem cells

21 January 2018

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Cord blood expansion. Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal

21 January 2018

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Wnt signaling controls the specification of definitive and primitive hematopoiesis from human pluripotent stem cells

21 January 2018

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Reprogramming committed murine blood cells to induced hematopoietic stem cells with defined factors

21 January 2018

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Reprogramming human endothelial cells to haematopoietic cells requires vascular induction

21 January 2018

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Stem cells: Reprogramming finds its niche

21 January 2018

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Gene therapy: Repair and replace

21 January 2018

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Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

21 January 2018

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Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

21 January 2018

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Targeted genome editing in human repopulating haematopoietic stem cells

21 January 2018

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Breaking the code of DNA binding specificity of TAL-type III effectors

21 January 2018

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Genome editing. The new frontier of genome engineering with CRISPR-Cas9

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.1111/CGE.12576

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2 September 2017

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PubMed ID

25708106