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Peptide Nucleic Acid Complex with Endosome Escape Ability and Uses thereof

机译:具有内体逃逸能力的肽核酸复合物及其用途

摘要

The present invention relates to a nucleic acid complex having a novel structure which can introduce a bioactive nucleic acid into a cell, a composition for the treatment or diagnosis of a disease comprising the same, and a method of regulating the expression of a target gene using the same. More specifically, the present invention relates to: a nucleic acid complex in which a bioactive nucleic acid containing a material that promotes endosomal escape and a carrier peptide nucleic acid are bonded in a complementary fashion; a composition for the treatment or diagnosis of a disease comprising the same; a composition for regulating the expression of a target gene; and a method of regulating the expression of a target gene using the same. The nucleic acid complex comprising a bioactive nucleic acid and a carrier peptide nucleic acid according to structural formula (1), [mA ≡ mC^(+)] can increase the stability of the bioactive nucleic acid; can reduce a loss such as precipitation due to self-aggregation of the bioactive nucleic acid, etc.; can increase delivery efficiency of the bioactive nucleic acid into a cell; and can conveniently regulate the expression of a target gene. Furthermore, the nucleic acid complex, due to containing the material promoting endosomal escape, is useful for enabling the nucleic acid complex having invaded into the cell through cell internalization to exhibit effective activities in cytoplasm.
机译:本发明涉及具有可将生物活性核酸引入细胞的新型结构的核酸复合物,用于治疗或诊断疾病的组合物以及使用该核酸复合物调节靶基因表达的方法。相同。更具体地,本发明涉及一种核酸复合物,其中含有促进内体逃逸的物质的生物活性核酸和载体肽核酸以互补方式结合。用于治疗或诊断疾病的组合物,包括该组合物;用于调节靶基因表达的组合物;以及使用该基因调节靶基因表达的方法。根据结构式(1)的包含生物活性核酸和载体肽核酸的核酸复合物,[mA≡mC^(+)]可以提高生物活性核酸的稳定性;可以减少由于生物活性核酸的自聚集等造成的沉淀等损失;可以提高生物活性核酸向细胞的递送效率;并且可以方便地调节靶基因的表达。此外,核酸复合物由于含有促进内体逃逸的物质,因此可用于使通过细胞内化作用侵入细胞内的核酸复合物在细胞质中发挥有效的活性。

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