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Peptide Nucleic Acid Complex with Endosome Escape Ability and Uses thereof
Peptide Nucleic Acid Complex with Endosome Escape Ability and Uses thereof
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机译:具有内体逃逸能力的肽核酸复合物及其用途
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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.
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