AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy.
作者:
主题词
动物(Animals);依赖病毒(Dependovirus);疾病模型, 动物(Disease Models, Animal);减量调节(Down-Regulation);女(雌)性(Female);基因表达调控(Gene Expression Regulation);基因沉默(Gene Silencing);遗传载体(Genetic Vectors);人类(Humans);注射, 静脉内(Injections, Intravenous);小鼠(Mice);小鼠, 近交C57BL(Mice, Inbred C57BL);小鼠, 转基因(Mice, Transgenic);微丝蛋白质类(Microfilament Proteins);肌, 骨骼(Muscle, Skeletal);肌营养不良, 面肩肱型(Muscular Dystrophy, Facioscapulohumeral);核蛋白质类(Nuclear Proteins);表型(Phenotype);RNA, 小分子干扰(RNA, Small Interfering);RNA结合蛋白质类(RNA-Binding Proteins);时间因素(Time Factors);转导, 遗传(Transduction, Genetic)
DOI
10.1038/mt.2011.153
PMID
21829175
发布时间
2021-10-20
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Molecular therapy
2055-64页
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