Expansion of genetically corrected neutrophils in chronic granulomatous disease mice by cotransferring a therapeutic gene and a selective amplifier gene View Full Text


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Article Info

DATE

2004-07-01

AUTHORS

T Hara, A Kume, Y Hanazono, H Mizukami, T Okada, H Tsurumi, H Moriwaki, Y Ueda, M Hasegawa, K Ozawa

ABSTRACT

Hematopoietic stem cell gene therapy has not provided clinical success in disorders such as chronic granulomatous disease (CGD), where genetically corrected cells do not show a selective advantage in vivo. To facilitate selective expansion of transduced cells, we have developed a fusion receptor system that confers drug-induced proliferation. Here, a ‘selective amplifier gene (SAG)’ encodes a chimeric receptor (GcRER) that generates a mitotic signal in response to estrogen. We evaluated the in vivo efficacy of SAG-mediated cell expansion in a mouse disease model of X-linked CGD (X-CGD) that is deficient in the NADPH oxidase gp91phox subunit. Bone marrow cells from X-CGD mice were transduced with a bicistronic retrovirus encoding GcRER and gp91phox, and transplanted to lethally irradiated X-CGD recipients. Estrogen was administered to a cohort of the transplants, and neutrophil superoxide production was monitored. A significant increase in oxidase-positive cells was observed in the estrogen-treated mice, and repeated estrogen administration maintained the elevation of transduced cells for 20 weeks. In addition, oxidase-positive neutrophils were increased in the X-CGD transplants given the first estrogen even at 9 months post-transplantation. These results showed that the SAG system would enhance the therapeutic effects by boosting genetically modified, functionally corrected cells in vivo. More... »

PAGES

1370-1377

References to SciGraph publications

  • 1990-11-01. High-Level Direct Expression of Semi-Synthetic Human Interleukin-6 in Escherichia coli and Production of N-Terminus Met-Free Product in BIO/TECHNOLOGY
  • 1996-08. Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells in NATURE MEDICINE
  • 2000-07-01. Long-term tracking of murine hematopoietic cells transduced with a bicistronic retrovirus containing CD24 and EGFP genes in GENE THERAPY
  • 2003-07-01. Occurrence of leukaemia following gene therapy of X-linked SCID in NATURE REVIEWS CANCER
  • 2000-09. In vivo selection using a cell-growth switch in NATURE GENETICS
  • 1995-02. Mouse model of X–linked chronic granulomatous disease, an inherited defect in phagocyte superoxide production in NATURE GENETICS
  • 2000-06. Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells in NATURE MEDICINE
  • 1999-06-01. Development of a modified selective amplifier gene for hematopoietic stem cell gene therapy in GENE THERAPY
  • 2002-11-01. Selective Expansion of Transduced Cells for Hematopoietic Stem Cell Gene Therapy in INTERNATIONAL JOURNAL OF HEMATOLOGY
  • 2002-07-01. Selective growth advantage of wild-type lymphocytes in X-linked SCID recipients in BONE MARROW TRANSPLANTATION
  • 2002-07-25. In vivo selective expansion of gene-modified hematopoietic cells in a nonhuman primate model in GENE THERAPY
  • 2002-04-01. Hematopoietic Stem Cell Gene Therapy in INTERNATIONAL JOURNAL OF HEMATOLOGY
  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/sj.gt.3302317

    DOI

    http://dx.doi.org/10.1038/sj.gt.3302317

    DIMENSIONS

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    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/15229634


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    38 schema:description Hematopoietic stem cell gene therapy has not provided clinical success in disorders such as chronic granulomatous disease (CGD), where genetically corrected cells do not show a selective advantage in vivo. To facilitate selective expansion of transduced cells, we have developed a fusion receptor system that confers drug-induced proliferation. Here, a ‘selective amplifier gene (SAG)’ encodes a chimeric receptor (GcRER) that generates a mitotic signal in response to estrogen. We evaluated the in vivo efficacy of SAG-mediated cell expansion in a mouse disease model of X-linked CGD (X-CGD) that is deficient in the NADPH oxidase gp91phox subunit. Bone marrow cells from X-CGD mice were transduced with a bicistronic retrovirus encoding GcRER and gp91phox, and transplanted to lethally irradiated X-CGD recipients. Estrogen was administered to a cohort of the transplants, and neutrophil superoxide production was monitored. A significant increase in oxidase-positive cells was observed in the estrogen-treated mice, and repeated estrogen administration maintained the elevation of transduced cells for 20 weeks. In addition, oxidase-positive neutrophils were increased in the X-CGD transplants given the first estrogen even at 9 months post-transplantation. These results showed that the SAG system would enhance the therapeutic effects by boosting genetically modified, functionally corrected cells in vivo.
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    50 bone marrow cells
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    53 cells
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    57 clinical success
    58 cohort
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    66 elevation
    67 estrogen
    68 estrogen administration
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