申请人:——
公开号:US20030207400A1
公开(公告)日:2003-11-06
The present invention pertains to novel products suitable for use as gene delivery systems in which nucleic acid is linked to a ligand in order to facilitate delivery of the nucleic acid to a target cell or sub-cellular compartment via uptake of the ligand. More particularly, the present invention pertains to vectors comprising: (a) a double stranded DNA (dsDNA) having at least one target sequence; and, (b) a chimeric molecule comprising: (i) a sequence specific polyamide (SSP) moiety bound non-covalently to said target sequence; and, (ii) a ligand moiety linked covalently to said sequence specific polyamide. The present invention also pertains to compositions comprising such chimeric molecules and vectors; methods for making such chimeric molecules and vectors; and methods of using such chimeric molecules and vectors, e.g., to deliver nucleic acid vectors to cells or sub-cellular compartments.
本发明涉及新型产品,适用于用作基因传递系统,其中核酸与配基连接,以便通过配基的摄取促进核酸传递到目标细胞或亚细胞区域。更具体地,本发明涉及包括:(a)至少具有一个目标序列的双链DNA(dsDNA);以及(b)包括的嵌合分子:(i)与所述目标序列非共价结合的序列特异性聚酰胺(SSP)基团;以及(ii)与所述序列特异性聚酰胺共价连接的配基基团。本发明还涉及包含这种嵌合分子和向量的组合物;制备这种嵌合分子和向量的方法;以及使用这种嵌合分子和向量的方法,例如将核酸向量传递到细胞或亚细胞区域。