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Peptide Nucleic Acid Complex with Endosome Escape Ability and Uses thereof

机译:肽核酸复合物与内体逃逸能力和用途

摘要

The present invention relates to a nucleic acid complex of a novel structure capable of introducing a bioactive nucleic acid into a cell, a composition for treating or diagnosing a disease comprising the same, and a method for regulating target gene expression using the same, and more particularly, to help endosomes escape It relates to a nucleic acid complex in which a bioactive nucleic acid containing a donor substance and a carrier peptide nucleic acid are complementarily combined, a composition for treating or diagnosing a disease containing the same, a composition for regulating target gene expression, and a method for regulating target gene expression using the same. The nucleic acid complex comprising the bioactive nucleic acid and the carrier peptide nucleic acid according to Structural Formula (1) of the present invention increases the stability of the bioactive nucleic acid and reduces the loss of precipitation due to self-aggregation of the bioactive nucleic acid. It is possible to increase the delivery efficiency of the bioactive nucleic acid in the cell, and to easily control the expression of the target gene. In addition, the nucleic acid complex is useful to include a substance that helps the endosomes escape, so that the nucleic acid complex penetrated into the cell by internalization effectively exhibits activity in the cytoplasm.
机译:本发明涉及一种能够将生物活性核酸引入细胞中的新型结构的核酸复合物,其用于治疗或诊断包含相同疾病的组合物,以及使用相同的靶基因表达的方法和更多特别是,为了帮助内染色体逸出,它涉及一种核酸络合物,其中含有供体物质和载体肽核酸的生物活性核酸互补地组合,一种用于治疗或诊断含有相同疾病的组合物,一种用于调节靶标的组合物基因表达,以及使用该方法调节靶基因表达的方法。根据本发明的结构式(1)的包含生物活性核酸和载体肽核酸的核酸络合物增加了生物活性核酸的稳定性,并降低了由于生物活性核酸的自聚集而降低沉淀的损失。可以提高细胞中生物活性核酸的输送效率,并容易地控制靶基因的表达。另外,核酸复合物可用于包括有助于内体逸出的物质,使得通过内化渗透到细胞中的核酸复合物有效地表现出细胞质中的活性。

著录项

  • 公开/公告号KR102262260B1

    专利类型

  • 公开/公告日2021-06-09

    原文格式PDF

  • 申请/专利权人

    申请/专利号KR1020180015750

  • 申请日2018-02-08

  • 分类号C12N15/11;A61K47/54;C07K14;C12Q1/6883;

  • 国家 KR

  • 入库时间 2022-08-24 19:15:17

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