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Invention Grant
CN106138050B Shh信号通路抑制剂的用途 失效 - 权利终止

Shh信号通路抑制剂的用途
Abstract:
本发明公开了一种Shh信号通路抑制剂的新用途。本发明所提供的Shh信号通路抑制剂的新用途具体为Shh信号通路抑制剂在制备治疗常染色体显性遗传性多囊肾(ADPKD)的产品中的应用；所述Shh信号通路抑制剂具体为Vismodegib。本发明以Vil‑Cre:Pkd2f3/f3小鼠作为ADPKD动物模型，结果表明Shh信号通路抑制剂—Vismodegib可减弱Vil‑Cre:Pkd2f3/f3小鼠的肾囊肿程度，可降低Vil‑Cre:Pkd2f3/f3小鼠的肾脏体重比，可降低Vil‑Cre:Pkd2f3/f3小鼠的血液中肌酐和尿素氮的含量；可降低Vil‑Cre:Pkd2f3/f3小鼠的肾脏纤维化程度。本发明对于ADPKD发病机制的分析，对于治疗ADPKD的药物的研发均具有重要意义。
Abstract(English):

The present invention discloses new uses of a Shh signaling pathway inhibitor. The new uses of the Shh signaling pathway inhibitor specifically comprises applications of the Shh signaling pathway inhibitor in preparation of autosomal domi-nant polycystic kidney disease (ADPKD) treatment product, wherein the Shh signaling pathway inhibitor is specifically Vismodegib. According to the present invention, a Vil-Cre:Pkd2f3/f3 mouse as an ADPKD animal model, and the results show that with the Shh signaling pathway inhibitor Vismodegib, the renal cyst degree of the Vil-Cre:Pkd2f3/f3 mouse can be reduced, the kidney weight ration of the Vil-Cre:Pkd2f3/f3 mouse can be reduced, the contents of creatinine and urea nitrogen in the blood of the Vil-Cre:Pkd2f3/f3 mouse can be reduced, and the renal fibrosis degree of the Vil-Cre:Pkd2f3/f3 mouse can be reduced; and the important significance is provided for the analysis of the ADPKD pathogenesis and the research and development of the ADPKD treatment drug.

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