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

Rodriguez-Gil, Jorge L; Baxter, Laura L; Watkins-Chow, Dawn E; Johnson, Nicholas L; Davidson, Cristin D; Carlson, Steven R; Incao, Arturo A; Wallom, Kerri L; Farhat, Nicole Y; Platt, Frances M; Dale, Ryan K; Porter, Forbes D; Pavan, William J

Rodriguez-Gil, Jorge L; Baxter, Laura L; Watkins-Chow, Dawn E; Johnson, Nicholas L; Davidson, Cristin D; Carlson, Steven R; Incao, Arturo A; Wallom, Kerri L; Farhat, Nicole Y; Platt, Frances M; Dale, Ryan K; Porter, Forbes D; Pavan, William J.

Afiliação

Rodriguez-Gil JL; Genomics, Development and Disease Section, Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Baxter LL; Medical Scientist Training Program, University of Wisconsin-Madison, School of Medicine and Public Health, Madison, WI 53705, USA.
Watkins-Chow DE; Genomics, Development and Disease Section, Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Johnson NL; Genomics, Development and Disease Section, Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Davidson CD; Bioinformatics and Scientific Programming Core, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA.
Carlson SR; Genomics, Development and Disease Section, Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Incao AA; Genomics, Development and Disease Section, Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Farhat NY; Department of Pharmacology, University of Oxford, Oxford OX1 3QT, UK.
Platt FM; Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA.
Dale RK; Department of Pharmacology, University of Oxford, Oxford OX1 3QT, UK.
Porter FD; Bioinformatics and Scientific Programming Core, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA.
Pavan WJ; Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA.

Hum Mol Genet ; 30(24): 2456-2468, 2021 11 30.

Article em En | MEDLINE | ID: mdl-34296265

Assuntos

Doença de Niemann-Pick Tipo C; 2-Hidroxipropil-beta-Ciclodextrina; Animais; Biomarcadores; Modelos Animais de Doenças; Proteínas do Olho/genética; Humanos; Glicoproteínas de Membrana/genética; Camundongos; Camundongos Knockout; Doença de Niemann-Pick Tipo C/tratamento farmacológico; Doença de Niemann-Pick Tipo C/genética; Doença de Niemann-Pick Tipo C/patologia; Transcriptoma

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Texto completo: 1 Coleções: 01-internacional Contexto em Saúde: 6_ODS3_enfermedades_notrasmisibles Base de dados: MEDLINE Assunto principal: Doença de Niemann-Pick Tipo C Limite: Animals / Humans Idioma: En Revista: Hum Mol Genet Ano de publicação: 2021 Tipo de documento: Article

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