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Macrophage models of Gaucher disease for evaluating disease pathogenesis and candidate drugs

Aflaki, Elma, Stubblefield, Barbara K., Maniwang, Emerson, Lopez, Grisel, Moaven, Nima, Goldin, Ehud, Marugan, Juan, Patnaik, Samarjit +5 more·2014·Science Translational Medicine
DOI 10.1126/scitranslmed.3008659PMC PMC4161206
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Abstract

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.

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Authors
Aflaki, E., Stubblefield, B. K., Maniwang, E., Lopez, G., Moaven, N., Goldin, E., Marugan, J., Patnaik, S., Dutra, A., Southall, N., Zheng, W., Tayebi, N., & Sidransky, E.
Affiliations
  • Section on Molecular Neurogenetics, Medical Genetics Branch, National Institutes of Health, Bethesda, MD 20892, USA.
  • National Center for Advancing Translational Sciences, National Institutes of Health, 9800 Medical Center Drive, Rockville, MD 20850, USA.
  • Cytogenetics and Microscopy Core, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Venue
Science Translational Medicine
Publisher
American Association for the Advancement of Science (AAAS)
Published
2014-06-11
Volume
6
Issue
240
ISSN
  • 1946-6234
  • 1946-6242
DOI
10.1126/scitranslmed.3008659
PMC
PMC4161206
Cited by
114
References
3
Language
en

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