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发明公开
CN110592082A 呼吸疾病相关基因特异性siRNA、含有siRNA的双螺旋寡RNA结构及其用途 审中-实审

呼吸疾病相关基因特异性siRNA、含有siRNA的双螺旋寡RNA结构及其用途
摘要：
本发明涉及与呼吸疾病相关的基因特异性siRNA，具体涉及与特发性肺纤维化和慢性阻塞性肺病(COPD)相关的基因特异性siRNA，以及含有所述基因特异性siRNA的高度有效的双螺旋寡RNA结构，其中所述双螺旋寡RNA结构具有如下结构，其中亲水性和疏水性材料用简单共价键或接头介导的共价键结合至双螺旋RNA结构(siRNA)的两端，从而有效转入细胞中，并且所述亲水性和疏水性材料能够通过溶液中双螺旋寡RNA结构的疏水作用转换为纳米颗粒。合意的是双螺旋寡RNA结构中含有的siRNA是对CTGF、Cyr61或Plekho1特异性siRNA，所述CTGF、Cyr61或Plekho1是与呼吸疾病尤其是特发性肺纤维化和COPD相关的基因。此外，本发明涉及用于产生双螺旋寡RNA结构的方法以及用于预防或治疗呼吸疾病(尤其是特发性肺纤维化和COPD)的含有双螺旋寡RNA结构的药物组合物。
摘要（英）：

The present invention relates to respiratory diseases, especially to gene specific siRNA related to idiopathic pulmonary fibrosis and chronic obstructive pulmonary disease (COPD), and a highly efficient double-helical oligo RNA structure containing the same, wherein the double-helical oligo RNA structure has a structure in which hydrophilic and hydrophobic materials are bonded at the both ends ofthe double-helical RNA (siRNA) using a simple covalent bond or a linker-mediated covalent bond to be effectively transferred into a cell, and is converted into nano particles by the hydrophobic interaction of the double-helical oligo RNA structure in a solution. It is desirable that the siRNA contained in the double-helical oligo RNA structure is a siRNA specific to a CTGF, Cyr61, or Plekho1, which are genes related to respiratory diseases, particularly idiopathic pulmonary fibrosis and COPD. In addition, the present invention relates to a method for producing the double-helical oligo RNA structure and a pharmaceutical composition containing the double-helical oligo RNA structure for preventing or treating respiratory diseases, particularly idiopathic pulmonary fibrosis and COPD.

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