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Down-regulation of gene expression using nucleic acid-carrying virus-like particles

机译:使用携带核酸的病毒样颗粒下调基因表达

摘要

Composition comprises a virus-like particle, which is built from more molecules of viral capsid proteins and contains a RNA-interference-inducing molecule. Independent claims are included for: (1) producing the composition, comprising assembling the viral capsid protein to virus-like protein in the presence of RNA-interference-inducing molecules; (2) introducing RNA-interference-inducing molecule into a target cell, comprising assembling the viral capsid protein to virus-like protein in the presence of the RNA-interference-inducing molecule, contacting the virus-like protein loaded with the RNA-interference-inducing molecules with the target cells under the condition in which an absorption of the RNA-interference-inducing molecules takes place into the target cells; (3) down regulation of at least a gene in the target cell, comprising providing the composition, contacting the composition with the target cell under the condition in which an absorption of the RNA-interference-inducing molecule takes place into the target cell; (4) a test kit for introducing the RNA-interference-inducing molecule into the target cells, comprising the composition; (5) a pharmaceutical composition comprising virus-like protein and RNA-interference-inducing molecule and optionally standard buffer, auxiliary materials, additives and/or diluting agents; (6) a modified polyoma JC virus-VP1 capsid protein comprising nuclear localization signal so that coding of nucleic acid or virus-like protein composition containing the modified capsid protein, takes place; (7) a virus-like protein composition, where the virus-like protein contains an immunostimulatory nucleic acid optionally in combination with polypeptide- or peptide-immunogen, an aptamer or a small interfering DNA molecule; and (8) a polyoma JC virus-VP1 capsid protein at which at least a heterologous binding partner e.g. a polypeptide, is bound over the amino acid residue lys-60 and/or lys-164. ACTIVITY : None given. MECHANISM OF ACTION : Gene therapy.
机译:组合物包含病毒样颗粒,该病毒样颗粒由更多的病毒衣壳蛋白分子构建而成,并包含RNA干扰诱导分子。包括以下独立权利要求:(1)生产该组合物,包括在存在RNA干扰诱导分子的情况下将病毒衣壳蛋白组装成病毒样蛋白;和(2)将RNA干扰诱导分子引入靶细胞,包括在RNA干扰诱导分子存在下将病毒衣壳蛋白组装成病毒样蛋白,使负载有RNA干扰的病毒样蛋白接触。在靶细胞中发生了RNA干扰诱导分子的吸收的条件下,与靶细胞一起诱导分子。 (3)下调靶细胞中至少一个基因的步骤,包括提供组合物,在使RNA干扰诱导分子吸收到靶细胞的条件下,使组合物与靶细胞接触; (4)用于将RNA干扰诱导分子导入靶细胞的试剂盒,其包含上述组合物。 (5)药物组合物,其包含病毒样蛋白和RNA干扰诱导分子以及任选的标准缓冲液,辅助材料,添加剂和/或稀释剂; (6)包含核定位信号的修饰的多瘤JC病毒-VP1衣壳蛋白,从而对包含修饰的衣壳蛋白的核酸或病毒样蛋白组合物进行编码。 (7)一种病毒样蛋白质组合物,其中所述病毒样蛋白质包含任选地与多肽或肽免疫原,适体或小的干扰DNA分子结合的免疫刺激性核酸; (8)一种多瘤JC病毒-VP1衣壳蛋白,在该蛋白上至少有一个异源结合伴侣,例如:多肽结合在氨基酸残基lys-60和/或lys-164上。活动:未提供。作用机理:基因治疗。

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