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    • 8. 发明申请
    • COMPLEXATION OF NUCLEIC ACIDS WITH DISULFIDE-CROSSLINKED CATIONIC COMPONENTS FOR TRANSFECTION AND IMMUNOSTIMULATION
    • 用转移和免疫接种的交联中和组分的核酸复合物
    • WO2012013326A1
    • 2012-02-02
    • PCT/EP2011/003719
    • 2011-07-25
    • CUREVAC GMBHBAUMHOF, PatrickVOSS, SöhnkeKRAMPS, ThomasKALLEN, Karl-Josef
    • BAUMHOF, PatrickVOSS, SöhnkeKRAMPS, ThomasKALLEN, Karl-Josef
    • C12N15/87A61K48/00A61K47/34A61K47/48
    • A61K48/0041A61K39/00A61K39/385A61K39/39A61K47/645A61K2039/51A61K2039/55516A61K2039/55561C12N15/87Y02A50/412Y02A50/472
    • The present invention is directed to a polymeric carrier cargo complex, comprising as a cargo at least one nucleic acid (molecule) and disulfide-crosslinked cationic components as a (preferably non-toxic and non-immunogenic) polymeric carrier. The inventive polymeric carrier cargo complex allows for both efficient transfection of nucleic acids into cells in vivo and in vitro and/or for induction of an (innate and/or adaptive) immune response, preferably dependent on the nucleic acid to be transported as a cargo. The present invention also provides, pharmaceutical compositions, particularly vaccines and adjuvants, comprising the inventive polymeric carrier cargo complex and optionally an antigen, as well as the use of such the inventive polymeric carrier cargo complex and optionally an antigen for transfecting a cell, a tissue or an organism, for (gene-)therapeutic purposes as disclosed herein, and/or as an immunostimulating agent or adjuvant, e.g. for eliciting an immune response for the treatment or prophylaxis of diseases as mentioned above. Finally, the invention relates to kits containing the inventive polymeric carrier cargo complex and/or the inventive pharmaceutical composition, adjuvant or vaccine in one or more parts of the kit.
    • 本发明涉及一种聚合物载体货物复合物,其包含至少一种核酸(分子)和作为(优选无毒的和非免疫原性的)聚合物载体的二硫键交联的阳离子组分。 本发明的聚合物载体货物复合物允许在体内和体外有效地将核酸转染到细胞中和/或诱导(先天和/或自适应)免疫应答,优选依赖于作为货物运输的核酸 。 本发明还提供药物组合物,特别是疫苗和佐剂,其包含本发明的聚合物载体货物复合物和任选的抗原,以及使用本发明的本发明的聚合物载体货物复合物和任选的抗原用于转染细胞,组织 或用于本文公开的(基因 - )治疗目的的生物体,和/或作为免疫刺激剂或佐剂,例如, 用于引发用于治疗或预防上述疾病的免疫应答。 最后,本发明涉及在试剂盒的一个或多个部分中含有本发明的聚合物载体货物复合物和/或本发明的药物组合物,佐剂或疫苗的试剂盒。