KIM, Hye Joo,김혜주,YU, Ji-Yeon,유지연,LEE, Dong In,이동인,KANG, Yusun,강유선,PARK, Hee Kyung,박희경
申请号:
KRKR2019/000245
公开号:
WO2019/156365A1
申请日:
2019.01.08
申请国别(地区):
KR
年份:
2019
代理人:
摘要:
The present invention relates to: a novel-structured nucleic acid complex for enabling a bioactive nucleic acid to be introduced into a cell; a composition comprising same, for treating or diagnosing a disease; and a method for regulating the expression of a target gene using same. More specifically, the present invention relates to: a nucleic acid complex containing an endosomal escape-aiding material, and having a bioactive nucleic acid complementarily bound with a carrier peptide nucleic acid; a composition comprising same, for treating or diagnosing a disease; a composition comprising same, for regulating the expression of a target gene; and a method for regulating the expression of a target gene using same. The nucleic acid complex, according to the present invention, represented by structural formula (1) and comprising the bioactive nucleic acid and the carrier peptide nucleic acid may increase the stability of the bioactive nucleic acid, reduce losses such as sedimentation due to the self-aggregation of the bioactive nucleic acid, increase efficiency in the delivery of the bioactive nucleic acid into a cell, and enable the expression of a target gene to be easily regulated. In addition, the nucleic acid complex contains the endosomal escape-aiding material, and thus is useful in enabling the nucleic acid complex to be effectively active at the cytoplasm when the nucleic acid complex has penetrated inside a cell by cellular internalization.La présente invention concerne : un complexe d'acide nucléique à nouvelle structure permettant d'introduire un acide nucléique bioactif dans une cellule ; une composition comprenant celui-ci, pour le traitement ou le diagnostic d'une maladie ; et un procédé pour la régulation de l'expression d'un gène cible utilisant celui-ci. Plus précisément, la présente invention concerne : un complexe d'acide nucléique contenant une substance favorisant l'échappement endosomal et ayant un acide nucléique bioactif lié de manière complémen