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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:肽核酸复合物与内体逃逸能力和用途
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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.
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