Adenoassociated virus serotype 9-mediated gene therapy for x-linked adrenoleukodystrophy.-论文-万方医学网

作者： Yi,Gong ^[1] ; Dakai,Mu ^[1] ; Shilpa,Prabhakar ^[1] ; Ann,Moser ^[2] ; Patricia,Musolino ^[1] ; JiaQian,Ren ^[1] ; Xandra O,Breakefield ^[1] ; Casey A,Maguire ^[1] ; Florian S,Eichler ^[3]

作者单位： Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA. ^[1] Peroxisome Disease Lab, Hugo W Moser Research Institute, Baltimore, Maryland, USA. ^[2] Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA. Electronic address: feichler@partners.org. ^[3]

主题词 ATP结合匣式转运子(ATP-Binding Cassette Transporters);肾上腺脑白质营养不良(Adrenoleukodystrophy);动物(Animals);脑(Brain);细胞系, 肿瘤(Cell Line, Tumor);细胞, 培养的(Cells, Cultured);依赖病毒(Dependovirus);疾病模型, 动物(Disease Models, Animal);脂肪酸类(Fatty Acids);成纤维细胞(Fibroblasts);基因表达(Gene Expression);基因, 报告(Genes, Reporter);遗传载体(Genetic Vectors);谷胱甘肽过氧化酶(Glutathione Peroxidase);人类(Humans);男(雄)性(Male);小鼠(Mice);小鼠, 基因敲除(Mice, Knockout);神经胶质(Neuroglia);蛋白质转运(Protein Transport);转导, 遗传(Transduction, Genetic)

DOI 10.1038/mt.2015.6

PMID 25592337

发布时间 2022-12-07

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Adenoassociated virus serotype 9-mediated gene therapy for x-linked adrenoleukodystrophy.

Molecular therapy

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Molecular therapy

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Adenoassociated virus serotype 9-mediated gene therapy for x-linked adrenoleukodystrophy.

Molecular therapy

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Molecular therapy

相关期刊

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