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    • 1. 发明授权
    • Sphingolipids polyalkylamine conjugates for use in transfection
    • 用于转染的鞘脂聚烷基胺共轭物
    • US08242089B2
    • 2012-08-14
    • US10560932
    • 2004-06-17
    • Yechezkel BarenholzDmitri Simberg
    • Yechezkel BarenholzDmitri Simberg
    • A61K48/00A61K9/127C12N15/00C12N15/88
    • A61K31/132A61K9/1272A61K38/47A61K39/39A61K48/0041A61K2039/543A61K2039/55555A61K2039/55561C07C271/20C12N15/88A61K2300/00
    • The present invention concerns the use of a sphingoid-polyalkylamine conjugate comprising a sphingoid backbone carrying, via a carbamoyl bond, at least one polyalkylamine chains as a capturing agent of nucleic acid molecules. The present invention also provides the use of the sphingoid polyalkylamine conjugate for the preparation of a pharmaceutical composition for the delivery of a nucleic acid molecule into a target cell. In a further aspect the invention provides a method for transfecting a nucleic acid into a target cell, the method comprises contacting said target cell with the sphingoid-polyalkylamine conjugate associated with a nucleic acid molecule, thereby transfecting said target cell with said nucleic acid molecule. Other aspects of the invention concern pharmaceutical compositions comprising said conjugate, therapeutic methods as well as kits, making use of the said conjugate. A preferred conjugate according to the invention is N-palmitoyl D-erythrosphingosyl-1-carbamoyl spermine.
    • 本发明涉及包含通过氨基甲酰键携带至少一个聚烷基胺链作为核酸分子的捕获剂的鞘氨醇主链的鞘氨醇 - 多烷基胺缀合物的用途。 本发明还提供了鞘氨醇多烷基胺缀合物用于制备用于将核酸分子递送至靶细胞的药物组合物的用途。 在另一方面,本发明提供了将核酸转染到靶细胞中的方法,所述方法包括使所述靶细胞与与核酸分子相关联的类鞘氨醇 - 多烷基胺缀合物接触,由此用所述核酸分子转染所述靶细胞。 本发明的其它方面涉及包含所述缀合物的药物组合物,治疗方法以及使用所述缀合物的试剂盒。 根据本发明的优选的缀合物是N-棕榈酰基D-赤藻酰基-1-氨基甲酰基精胺。
    • 4. 发明申请
    • Sphingolipids polyalkylamine conjugates for use in transfection
    • 用于转染的鞘脂聚烷基胺共轭物
    • US20060252718A1
    • 2006-11-09
    • US10560932
    • 2004-06-17
    • Yechezkel BarenholzDmitri Simberg
    • Yechezkel BarenholzDmitri Simberg
    • A61K48/00A61K9/127C12N15/88
    • A61K31/132A61K9/1272A61K38/47A61K39/39A61K48/0041A61K2039/543A61K2039/55555A61K2039/55561C07C271/20C12N15/88A61K2300/00
    • The present invention concerns the use of a sphingoid-polyalkylamine conjugate comprising a sphingoid backbone carrying, via a carbamoyl bond, at least one polyalkylamine chains as a capturing agent of nucleic acid molecules. The present invention also provides the use of the sphingoid polyalkylamine conjugate for the preparation of a pharmaceutical composition for the delivery of a nucleic acid molecule into a target cell. In a further aspect the invention provides a method for transfecting a nucleic acid into a target cell, the method comprises contacting said target cell with the sphingoid-polyalkylamine conjugate associated with a nucleic acid molecule, thereby transfecting said target cell with said nucleic acid molecule. Other aspects of the invention concern pharmaceutical compositions comprising said conjugate, therapeutic methods as well as kits, making use of the said conjugate. A preferred conjugate according to the invention is N-palmitoyl D-erythrosphingosyl-1-carbamoyl spermine.
    • 本发明涉及包含通过氨基甲酰键携带至少一个聚烷基胺链作为核酸分子的捕获剂的鞘氨醇主链的鞘氨醇 - 多烷基胺缀合物的用途。 本发明还提供了鞘氨醇多烷基胺缀合物用于制备用于将核酸分子递送至靶细胞的药物组合物的用途。 在另一方面,本发明提供了将核酸转染到靶细胞中的方法,所述方法包括使所述靶细胞与与核酸分子相关联的类鞘氨醇 - 多烷基胺缀合物接触,由此用所述核酸分子转染所述靶细胞。 本发明的其它方面涉及包含所述缀合物的药物组合物,治疗方法以及使用所述缀合物的试剂盒。 根据本发明的优选的缀合物是N-棕榈酰基D-赤藻酰基-1-氨基甲酰基精胺。