Macrophage Models of Gaucher Disease for Evaluating Disease Pathogenesis and Candidate Drugs-Reference-Cited by-同舟云学术

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

Published:2014-06-11 Issue:240 Volume:6 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Aflaki Elma¹,Stubblefield Barbara K.¹,Maniwang Emerson¹,Lopez Grisel¹,Moaven Nima¹,Goldin Ehud¹,Marugan Juan²,Patnaik Samarjit²,Dutra Amalia³,Southall Noel²,Zheng Wei²,Tayebi Nahid¹,Sidransky Ellen¹

Affiliation:

1. Section on Molecular Neurogenetics, Medical Genetics Branch, National Institutes of Health, Bethesda, MD 20892, USA.

2. National Center for Advancing Translational Sciences, National Institutes of Health, 9800 Medical Center Drive, Rockville, MD 20850, USA.

3. Cytogenetics and Microscopy Core, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.

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.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference38 articles.

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2. Glucosylsphingosine accumulation in tissues from patients with Gaucher disease: correlation with phenotype and genotype

3. The fine structure of the cerebroside occurring in Gaucher's disease.

4. Multicenter Analysis of Glucocerebrosidase Mutations in Parkinson's Disease

5. Cell-Surface Calreticulin Initiates Clearance of Viable or Apoptotic Cells through trans-Activation of LRP on the Phagocyte

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