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

在本研究中，由于育种方面的问题，Pkd2基因缺失突变诱导的转基因medaka的功效评价体系未能建立。然而，在干扰素诱导的Pkd1条件敲除小鼠中，在出生后一周开始干扰素作为早期囊肿形成组，观察到明显的囊肿形成。在这些Pkd1条件敲除小鼠中，可以评估mTOR抑制剂对囊性形成的功效。提示该小鼠可作为多囊肾病治疗疗效评价体系的模型动物之一。

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.