首页> 外国专利> AMPHIREGULIN-SPECIFIC DOUBLE-HELICAL OLIGO-RNA, DOUBLE-HELICAL OLIGO-RNA STRUCTURE COMPRISING DOUBLE-HELICAL OLIGO-RNA, AND COMPOSITION FOR PREVENTING OR TREATING RESPIRATORY DISEASES CONTAINING SAME

AMPHIREGULIN-SPECIFIC DOUBLE-HELICAL OLIGO-RNA, DOUBLE-HELICAL OLIGO-RNA STRUCTURE COMPRISING DOUBLE-HELICAL OLIGO-RNA, AND COMPOSITION FOR PREVENTING OR TREATING RESPIRATORY DISEASES CONTAINING SAME

机译:两性调节素特定的双螺旋寡RNA,包含双螺旋寡RNA的双螺旋寡RNA结构以及用于预防或治疗包含相同疾病的呼吸系统疾病的成分

摘要

The present invention relates to an amphiregulin-specific double-stranded oligo siRNA, which can inhibit the expression of amphiregulin with high efficiency and can be effectively used for the prevention or treatment of respiratory diseases. Moreover, the present invention relates to an amphiregulin-specific double-stranded oligo siRNA structure that comprises hydrophilic and hydrophobic compounds bound to the amphiregulin-specific double-stranded oligo siRNA by a simple covalent bond or a linker-mediated covalent bond so as to increase the in vivo stability and intracellular delivery efficiency of the double-stranded oligo siRNA, and to nanoparticles composed of the oligo siRNA structures. The amphiregulin-specific double-stranded oligo siRNA structure according to the present invention can increase the stability of the amphiregulin-specific double-stranded oligo siRNA without impairing the function of the amphiregulin-specific double-stranded oligo siRNA, and at the same time, can efficiently increase the membrane permeability of the double-stranded oligo siRNA. Thus, when the amphiregulin-specific double-stranded oligo siRNA structure is used, the amphiregulin-specific double-stranded oligo siRNA can be efficiently delivered into cells even when it is administered at a relatively low concentration, so that it can be effectively used for the prevention or treatment of respiratory diseases.
机译:本发明涉及一种双调蛋白特异性双链寡核苷酸siRNA,其可以高效抑制双调蛋白的表达,并且可以有效地用于预防或治疗呼吸系统疾病。此外,本发明涉及一种双调蛋白特异性双链寡siRNA结构,其包含通过简单的共价键或接头介导的共价键与双调蛋白特异性双链寡siRNA结合的亲水和疏水化合物,从而增加双链寡核苷酸siRNA的体内稳定性和细胞内递送效率,以及由寡核苷酸siRNA结构组成的纳米颗粒。根据本发明的双调蛋白特异性双链寡siRNA结构可以在不损害双调蛋白特异性双链寡siRNA的功能的情况下增加双调蛋白特异性双链寡siRNA的稳定性,同时,可以有效提高双链寡聚siRNA的膜通透性。因此,当使用双调蛋白特异性双链寡siRNA结构时,即使以相对低的浓度施用,也可以有效地将双调蛋白特异性双链寡siRNA有效地递送到细胞中,从而可以有效地用于预防或治疗呼吸系统疾病。

著录项

  • 公开/公告号EP2905337A4

    专利类型

  • 公开/公告日2016-10-12

    原文格式PDF

  • 申请/专利权人 BIONEER CORPORATION;

    申请/专利号EP20130843342

  • 发明设计人 PARK HAN-OH;CHAE JEIWOOK;YOON PYOUNG OH;

    申请日2013-10-07

  • 分类号C12N15/113;A61K31/7105;

  • 国家 EP

  • 入库时间 2022-08-21 14:53:10

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