公开(公告)号：WO01076633A3

公开(公告)日：2002-02-14

申请号：PCT/EP2001/003746

申请日：2001-04-03

IPC: A61K47/48 ,  C12N15/87

CPC classification number: B82Y5/00 ,  A61K47/645 ,  A61K47/6898 ,  C08G83/004 ,  C12N15/87

Abstract: The invention relates to a method for carrying out the targeted transfection of cells, to compositions, which can be used for such methods, and to corresponding medicaments for use in gene therapy. The invention particularly relates to a method for introducing nucleic acid into cells involving the following steps: (a) mixing a nucleic acid with a dendrimer, whereby a portion of the dendrimer molecules is biotinylated; mixing the prepared complex, which consists of a nucleic acid and a dendrimer, with a second complex, which consists of an avidin or a streptavidin and of a biotinylated target-specific binding molecule, and; (c) incubating the complex prepared in step (b) with cells. Dendrimers that are well-suited for the invention are, for example, partially solvolyzed polyamidoamine (PAMAM) dendrimers. Target-specific binding molecules are, in particular, cell type-specific markers also of the cell surface of the target cells.

Abstract translation: 本发明涉及一种用于细胞的靶向的转染方法，一种能够被用于这样的程序，以及用于基因治疗的相应的药物组合物。 特别地，本发明涉及一种用于将核酸导入包括以下步骤的细胞：（a）混合核酸与树枝状聚合物，其特征在于树枝状聚合物分子的比例是生物素化; （B）混合所得的核酸和树枝状聚合物用由抗生物素蛋白或链霉亲和生物素化的靶标特异性结合分子的第二复合物的复合物; 和（c）在步骤（b）温育的细胞所得到的复合物。 例如部分solvolysierte是聚酰胺胺（PAMAM）树枝状聚合物，本发明非常适合于树枝状聚合物。 靶特异性结合分子是特定细胞类型特异性标志物和靶细胞的细胞表面。