Macrophage Models of Gaucher Disease for Evaluating Disease Pathogenesis and Candidate Drugs

  • Elma Aflaki
    Section on Molecular Neurogenetics, Medical Genetics Branch, National Institutes of Health, Bethesda, MD 20892, USA.
  • Barbara K. Stubblefield
    Section on Molecular Neurogenetics, Medical Genetics Branch, National Institutes of Health, Bethesda, MD 20892, USA.
  • Emerson Maniwang
    Section on Molecular Neurogenetics, Medical Genetics Branch, National Institutes of Health, Bethesda, MD 20892, USA.
  • Grisel Lopez
    Section on Molecular Neurogenetics, Medical Genetics Branch, National Institutes of Health, Bethesda, MD 20892, USA.
  • Nima Moaven
    Section on Molecular Neurogenetics, Medical Genetics Branch, National Institutes of Health, Bethesda, MD 20892, USA.
  • Ehud Goldin
    Section on Molecular Neurogenetics, Medical Genetics Branch, National Institutes of Health, Bethesda, MD 20892, USA.
  • Juan Marugan
    National Center for Advancing Translational Sciences, National Institutes of Health, 9800 Medical Center Drive, Rockville, MD 20850, USA.
  • Samarjit Patnaik
    National Center for Advancing Translational Sciences, National Institutes of Health, 9800 Medical Center Drive, Rockville, MD 20850, USA.
  • Amalia Dutra
    Cytogenetics and Microscopy Core, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
  • Noel Southall
    National Center for Advancing Translational Sciences, National Institutes of Health, 9800 Medical Center Drive, Rockville, MD 20850, USA.
  • Wei Zheng
    National Center for Advancing Translational Sciences, National Institutes of Health, 9800 Medical Center Drive, Rockville, MD 20850, USA.
  • Nahid Tayebi
    Section on Molecular Neurogenetics, Medical Genetics Branch, National Institutes of Health, Bethesda, MD 20892, USA.
  • Ellen Sidransky
    Section on Molecular Neurogenetics, Medical Genetics Branch, National Institutes of Health, Bethesda, MD 20892, USA.

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説明

<jats:p>Macrophages differentiated from monocytes or induced pluripotent stem cells derived from patients with Gaucher disease facilitate investigation of disease pathogenesis and validation of new candidate drugs.</jats:p>

収録刊行物

  • Science Translational Medicine

    Science Translational Medicine 6 (240), 240ra273-, 2014-06-11

    American Association for the Advancement of Science (AAAS)

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