首页> 外国专利> BIOLOGICALLY ACTIVE MOLECULES THAT ARE BASED ON SIRNA AND THAT CAN BE ACTIVATED IN A CELL-SPECIFIC MANNER, METHOD FOR ACTIVATING SAME, AND APPLICATION KIT FOR ADMINISTRATION

BIOLOGICALLY ACTIVE MOLECULES THAT ARE BASED ON SIRNA AND THAT CAN BE ACTIVATED IN A CELL-SPECIFIC MANNER, METHOD FOR ACTIVATING SAME, AND APPLICATION KIT FOR ADMINISTRATION

机译:基于SIRNA的生物活性分子,可以特定于细胞的方式被激活,用于激活相同分子的方法以及用于管理的应用工具包

摘要

The aim of the invention was to create biological active substances that can be transfected into or onto target cells in living beings, plants, and fungi in vitro as well as in vivo and only there inhibit the expression of genes highly effectively without affecting the target-gene expression specific to the active agent and thus the formation of proteins in other cells of the organism. According to the invention, the siRNA (1) has at least one covalent bond (6) to one or more oligosaccharides (3), which each have one or more specific chemical structures selected for enzymes typical of the target cells. By means of said bond (6), which is broken only by said enzyme or enzymes typical of the target cells in order to biologically activate the active substance, the molecules remain biologically inactive in cells other than the target cells. Said molecules are used, for example, to influence the gene expression of preferably diseased organs, cells, or plants that are infected and afflicted with parasites or fungi.
机译:本发明的目的是创造可以在体外以及在体内转染到生物,植物和真菌的靶细胞内或上的生物活性物质。只有在那里有效地抑制基因表达,而不影响活性剂特异的靶基因表达,从而不影响生物体其他细胞中蛋白质的形成。根据本发明,siRNA(1)具有与一种或多种寡糖(3)的至少一个共价键(6),其各自具有针对靶细胞典型的酶选择的一种或多种特异性化学结构。通过所述键(6),所述键(6)仅被所述靶细胞典型的一种或多种酶破坏以便生物活化活性物质,分子在靶细胞以外的细胞中保持生物惰性。所述分子例如用于影响优选被感染并患有寄生虫或真菌的患病器官,细胞或植物的基因表达。

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