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    • 8. 发明申请
    • COMPOSITIONS AND METHODS FOR THE DELIVERY OF BIOLOGICALLY ACTIVE RNAS
    • 用于递送生物活性RNAs的组合物和方法
    • WO2010105277A1
    • 2010-09-16
    • PCT/US2010/027365
    • 2010-03-15
    • EGEN, INC.POLACH, KevinFEWELL, JasonANWER, Khursheed
    • POLACH, KevinFEWELL, JasonANWER, Khursheed
    • C12N15/63A61K31/7052
    • C12N15/113C12N15/63C12N15/85C12N15/87
    • The present invention provides novel compounds, compositions, and methods for the delivery of biologically active RNA molecules to cells. Specifically, the invention provides novel nucleic acid molecules, polypeptides, and RNA-protein complexes useful for the delivery of biologically active RNAs to cells and polynucleotides encoding the same. The invention also provides vectors for expressing said polynucleotides. In addition, the invention provides cells and compositions comprising the novel compounds and vectors, which can be used as transfection reagents. The invention further provides methods for producing said compounds, vectors, cells, and compositions. Additionally, vectors and methods for delivering biologically active RNA molecules to cells and/or tissues are provided. The novel compounds, vectors, cells, and compositions are useful, for example, in delivering biologically active RNA molecules to cells to modulate target gene expression in the diagnosis, prevention, amelioration, and/or treatment of diseases, discorders, or conditions in a subject or organism.
    • 本发明提供了用于将生物活性RNA分子递送至细胞的新型化合物,组合物和方法。 具体地说,本发明提供了可用于将生物活性RNA递送到细胞的新型核酸分子,多肽和RNA-蛋白复合物,以及编码该核酸的多核苷酸。 本发明还提供用于表达所述多核苷酸的载体。 此外,本发明提供了可用作转染试剂的包含新化合物和载体的细胞和组合物。 本发明还提供了生产所述化合物,载体,细胞和组合物的方法。 另外,提供了将生物活性RNA分子递送到细胞和/或组织的载体和方法。 新型化合物,载体,细胞和组合物可用于例如将生物活性RNA分子递送至细胞以调节靶基因表达在诊断,预防,改善和/或治疗疾病,不便者或疾病中的病症 主体或生物体。