常染色体显性多囊肾病是一种单基因遗传性疾病，是我国引起终末期肾病的四大病因之一。开发缓解或治疗多囊肾的有效药物目前是医学界的一大难题。研究发现，自噬诱导剂可以缓解多囊肾疾病的发生和发展。而苦参碱是一种新型细胞自噬诱导剂。故本研究假设：苦参碱可通过诱导自噬治疗或缓解多囊肾疾病。前期细胞实验表明：用苦参碱干预多囊肾细胞PH2和IMCD3后，细胞自噬明显增强；初步动物实验结果表明：苦参碱能明显降低小鼠肾囊肿指数，延长模型小鼠的存活时间。据此，本项目将使用慢性和急性多囊肾小鼠模型进一步证实苦参碱对多囊肾的干预作用；通过肾囊肿指数研究苦参碱治疗多囊肾性效果；通过RT-PCR和Western-blot等分子生物学技术，基于蛋白质组和转录组水平，研究苦参碱对细胞自噬和肾囊肿形成的相关信号途径。本项目研究传统中医药治疗多囊肾的可能性，为其治疗药物的研发提供新的资源。

Autosomal dominant polycystic kidney disease (ADPKD) is a monogenic hereditary disease， which is one of the four major causes of end-stage renal disease in China. It is a world difficult problem to find good medicine on ADPKD. Treatment with autophagy inducers could significantly alleviate the development of renal cysts. It has been found that matrine can induce autophagy and is a new autophagy inducer. Previous cell experiments showed that the autophagy of PH2 and IMCD3，the model cell of polycystic kidney， was significantly enhanced after matrine intervention. Animal model experiments showed that matrine significantly reduced the index of renal cyst in mice and prolonged the survival time of model mice. Accordingly, this project will use mouse model Pkd1flox/flox:Tamoxifen-Cre and Pkd1flox/flox:Pkhd1-cre to further confirm the therapeutic effect of matrine on polycystic kidney disease; through RT-PCR and Western-blot molecular biology technology, to study the effect of matrine on related signal pathways of autophagy and cystogenesis based on proteome and transcriptome level. Finally, we will elucidate the signal pathways related to matrine intervention in renal cyst formation and autophagy, and determine the clinical application prospect of matrine in polycystic kidney. This project will study the possibility of traditional Chinese medicine（TCM） on the treatment of polycystic kidney disease, and provides a new resource for the development of ADPKD therapeutic drugs.