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    • 7. 发明申请
    • TRANSCRIPTIONAL REGULATION OF GENE EXPRESSION BY SMALL DOUBLE-STRANDED MODULATORY RNA
    • 小双链调控RNA对基因表达的转录调控
    • WO2005001110A2
    • 2005-01-06
    • PCT/US2004017208
    • 2004-05-28
    • SALK INST FOR BIOLOGICAL STUDIGAGE FRED HKUWABARA TOMOKO
    • GAGE FRED HKUWABARA TOMOKO
    • A61K48/00C12N5/0793C12N15/867C12Q20060101C12Q
    • C12N5/0619C12N2501/60C12N2799/027
    • The invention provides a method for modulating gene expression by contacting a cellular system with a double-stranded ribonucleic acid molecule capable of associating with a regulatory machinery that controls transcription of one or more genes, wherein the association results in altered expression of the one or more genes. The invention is further directed to a method for directing the differentiation of neuronal stem cells into neurons by contacting a cellular system with a double-stranded ribonucleic acid molecule capable of associating with a regulatory machinery that controls transcription of one or more genes involved in neuronal differentiation and directing the transcription of the one or more genes. In related embodiments, the invention provides particular compositions of double-stranded ribonucleic acid molecules as well as therapeutic and screening applications of the invention.
    • 本发明提供了通过使细胞系统与双链核糖核酸分子接触来调节基因表达的方法,所述双链核糖核酸分子能够与控制一种或多种基因的转录的调节机制相关联,其中所述结合导致一种或多种 基因。 本发明还涉及通过使细胞系统与双链核糖核酸分子接触来指导神经元干细胞分化为神经元的方法,所述双链核糖核酸分子能够与控制参与神经元分化的一种或多种基因的转录的调节机制 并指导一个或多个基因的转录。 在相关的实施方案中,本发明提供了双链核糖核酸分子的特定组合物以及本发明的治疗和筛选应用。