Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease. (Q45875985)

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scientific article published in June 2004

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English	Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease.	scientific article published in June 2004

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29 December 2019

Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease (English)

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29 December 2019

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29 December 2019

author name string

A Alex Hofling

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29 December 2019

Steven Devine

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29 December 2019

Carole Vogler

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1 June 2004

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29 December 2019

Molecular Therapy

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29 December 2019

856-865

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29 December 2019

Prevalence of lysosomal storage disorders

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.03.013

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Lysosomal storage diseases

1 reference

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Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors

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Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency

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Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis

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Abnormal osteoclast morphology and bone remodeling in a murine model of a lysosomal storage disease.

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Behavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII.

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Retinal function is improved in a murine model of a lysosomal storage disease following bone marrow transplantation

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Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation

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Gene-marking to trace origin of relapse after autologous bone-marrow transplantation.

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Results of MDR-1 vector modification trial indicate that granulocyte/macrophage colony-forming unit cells do not contribute to posttransplant hematopoietic recovery following intensive systemic therapy

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SCID mouse models of human stem cell engraftment

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Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID mouse bone marrow: implications for gene therapy

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Sustained proliferation, multi-lineage differentiation and maintenance of primitive human haemopoietic cells in NOD/SCID mice transplanted with human cord blood

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Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors

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Transduction of Human CD34+CD38- Bone Marrow and Cord Blood-Derived SCID-Repopulating Cells with Third-Generation Lentiviral Vectors

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Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient

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Third-generation, self-inactivating gp91(phox) lentivector corrects the oxidase defect in NOD/SCID mouse-repopulating peripheral blood-mobilized CD34+ cells from patients with X-linked chronic granulomatous disease

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Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice.

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7 January 2021

Engraftment of human CD34+ cells leads to widespread distribution of donor-derived cells and correction of tissue pathology in a novel murine xenotransplantation model of lysosomal storage disease.

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7 January 2021

A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII

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Potent interleukin 3 receptor agonist with selectively enhanced hematopoietic activity relative to recombinant human interleukin 3

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Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

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Optimization of gene transfer into primitive human hematopoietic cells of granulocyte-colony stimulating factor-mobilized peripheral blood using low-dose cytokines and comparison of a gibbon ape leukemia virus versus an RD114-pseudotyped retroviral

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Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood

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Differential transduction efficiency of SCID-repopulating cells derived from umbilical cord blood and granulocyte colony-stimulating factor-mobilized peripheral blood

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Lentiviral gene transfer into peripheral blood-derived CD34+ NOD/SCID-repopulating cells

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Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis

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A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

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