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    • 3. 发明申请
    • POLYAMIDES FOR NUCLEIC ACID DELIVERY
    • 用于核酸递送的聚酰胺
    • US20110183417A1
    • 2011-07-28
    • US13044969
    • 2011-03-10
    • Theresa M. Reineke
    • Theresa M. Reineke
    • C12N15/64
    • C12N15/113C12N2310/13C12N2310/51
    • The present invention provides a new class of non-viral transduction vectors that can be used for both in vivo and in vitro applications. In particular, these vectors can be used for gene transfer applications. These new gene transduction vectors can achieve transfer efficiencies far greater to commercially available polymeric and liposomal gene transfer vectors while maintaining little or no toxicity in vitro. Their low in vitro toxicity makes them ideal candidates for in vivo use. The present invention also provides a gene transfer vector that has comparable efficiency to a viral vector without the potential for a life-threatening immune response. Furthermore, the unique polycationic structure of these polymers associates with many suitable biologically active molecule, including oligonucleotides and polypeptides and other compounds that poses multiple cationic sites. The polymer can act as a delivery vehicle for the associated biologically active molecule, in vivo or in vitro, to the cells of interest for the biologically active molecule. Complexes according to the invention or portions thereof, can comprise a cellular delivery molecule or agent that can facilitate the translocation of the complex or portion thereof into cells. In some embodiments, cellular delivery molecules for use in the present invention may comprise one or more one or more polymers of the present invention, e.g., polyamides, dendritic macromolecules (polymers comprising an oligoamine shell and a cyclodextrin core), and carbohydrate-containing degradable polyesters.
    • 本发明提供了可用于体内和体外应用的新一类非病毒转导载体。 特别地,这些载体可用于基因转移应用。 这些新的基因转导载体可以实现远远大于商业上可获得的聚合物和脂质体基因转移载体的转移效率,同时在体外保持很少或没有毒性。 它们的低体外毒性使其成为体内使用的理想候选物。 本发明还提供了具有与病毒载体相当的效率而不具有危及生命的免疫应答潜力的基因转移载体。 此外,这些聚合物的独特的聚阳离子结构与许多合适的生物活性分子结合,包括寡核苷酸和多肽以及构成多个阳离子位点的其它化合物。 聚合物可以作为生物活性分子感兴趣的细胞在体内或体外作为相关生物活性分子的递送载体。 根据本发明或其部分的复合物可以包含可以促进复合物或其部分转运到细胞中的细胞递送分子或试剂。 在一些实施方案中,用于本发明的细胞递送分子可以包含本发明的一种或多种一种或多种聚合物,例如聚酰胺,树突状大分子(包含寡胺壳和环糊精核心的聚合物)和含碳水化合物的可降解 聚酯。
    • 5. 发明授权
    • Polyamides for nucleic acid delivery
    • 用于核酸输送的聚酰胺
    • US07927873B2
    • 2011-04-19
    • US10596522
    • 2004-12-20
    • Theresa M. Reineke
    • Theresa M. Reineke
    • C12N15/00A61K48/00A61K31/715A61K31/44
    • C12N15/113C12N2310/13C12N2310/51
    • The present invention provides a new class of non-viral transduction vectors that can be used for both in vivo and in vitro applications. The present invention also provides a gene transfer vector that has comparable efficiency to a viral vector without the potential for a life-threatening immune response. Complexes according to the invention or portions thereof, can comprise a cellular delivery molecule or agent that can facilitate the translocation of the complex or portion thereof into cells. In some embodiments, cellular delivery molecules for use in the present invention may comprise one or more polymers of the present invention, e.g., polyamides, dendritic macromolecules and carbohydrate-containing degradable polyesters.
    • 本发明提供了可用于体内和体外应用的新一类非病毒转导载体。 本发明还提供了具有与病毒载体相当的效率而不具有危及生命的免疫应答潜力的基因转移载体。 根据本发明或其部分的复合物可以包含可以促进复合物或其部分转运到细胞中的细胞递送分子或试剂。 在一些实施方案中,用于本发明的细胞递送分子可以包含一种或多种本发明的聚合物,例如聚酰胺,树枝状大分子和含碳水化合物的可降解聚酯。