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发明公开
CN106906213A 一种修饰的siRNA及其用途 失效 - 权利终止

一种修饰的siRNA及其用途
摘要：
本发明公开了一种修饰的siRNA及用途。本发明以吉西他滨为siRNA修饰单体为例，对靶向RRM1mRNA的siRNA进行特异性地有效修饰，获得一种新型的小分子靶向化合物Gem‑RRM1siRNA，不仅能有效抑制胰腺癌细胞增殖，还能增强RRM1siRNA逆转吉西他滨耐药胰腺癌细胞的作用，起到协同抑制肿瘤的效果。可以预见：具有生物活性或细胞毒性的核苷类似物应当也可以作为siRNA修饰单体，对特定的siRNA进行特异性地有效修饰后，二者起到协同作用，可应用于疾病治疗药物开发和制备。
摘要（英）：

The invention discloses a modified siRNA and application thereof. Gemcitabine as an siRNA modified monomer is taken as an example, specific effective modification is carried out on siRNA of targeted RRM1mRNA, and a novel small-molecularly targeted compound Gem-RRM1siRNA is obtained, so that the cell proliferation of pancreatic cancer can be effectively inhibited, the effect of reversing gemcitabine drug-resistant pancreatic cancer cells by the RRM1siRNA can also be strengthened, and the effect of synergistic tumor inhibition is achieved. The following is foreseeable: nucleoside analogs with biological activity or cell toxicity also can serve as the siRNA modified monomer, and after the specific effective modification is carried out on the specific siRNA, a synergistic effect is achieved, so that the modified siRNA can be applied to disease treatment medicine development and preparation.

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