Generation of animal models and establishment of the drug-efficacy evaluation system in polycystic kidney disease.

在本研究中，由于育种上的问题，未能建立PKD2基因缺失突变转基因青竹的药效评价体系。然而，在干扰素诱导的条件基因敲除小鼠中，干扰素在出生后一周开始作为早期包囊形成组，观察到显著的包囊形成。可以评估mTOR抑制剂对这些PKD1条件性基因敲除小鼠的囊性形成的疗效。这些小鼠有望成为治疗多囊肾病疗效评价系统的模型动物之一。

In this study, the efficacy evaluation system in the transgenic medaka induced by deletion mutation of Pkd2 gene was not able to be established because of the problem of the breeding. However, in interferon-inducible Pkd1 conditional knockout mice, in which interferon was started at one week after birth as an early cyst formation group, remarkable cyst formation was observed. It was possible to evaluate the efficacy of mTOR inhibitor for cystic formation in these Pkd1 conditional knockout mice. It was suggested that these mice would be one of the model animals which could perform an efficacy evaluation system for treatment in polycystic kidney diseases.