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Transcriptome of HPβCD-treated Niemann-Pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics.

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第一作者： Jorge L,Rodriguez-Gil

第一单位： Genomics, Development and Disease Section, Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.;Medical Scientist Training Program, University of Wisconsin-Madison, School of Medicine and Public Health, Madison, WI 53705, USA.

作者： Jorge L,Rodriguez-Gil ^[1] ; Laura L,Baxter ^[2] ; Dawn E,Watkins-Chow ^[2] ; Nicholas L,Johnson ^[3] ; Cristin D,Davidson ^[2] ; Steven R,Carlson ^[2] ; Arturo A,Incao ^[2] ; NISC Comparative Sequencing Program ^[4] ; Kerri L,Wallom ^[5] ; Nicole Y,Farhat ^[4] ; Frances M,Platt ^[3] ; Ryan K,Dale ^[5] ; Forbes D,Porter ^[2] ; William J,Pavan

作者单位： Genomics, Development and Disease Section, Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.;Medical Scientist Training Program, University of Wisconsin-Madison, School of Medicine and Public Health, Madison, WI 53705, USA. ^[1] Genomics, Development and Disease Section, Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA. ^[2] Bioinformatics and Scientific Programming Core, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA. ^[3] Department of Pharmacology, University of Oxford, Oxford OX1 3QT, UK. ^[4] Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA. ^[5]