中文摘要：

常染色体显性多囊肾病（ADPKD）是一种常见的遗传性肾脏病，以双肾多发液性囊肿为特征。ADPKD患病率约1／1000，是尿毒症的第四位病因。大约有50％的多囊肾患者在60岁以前会进入终末期肾病阶段，缺乏特效的治疗措施。近年来，ADPKD分子机制研究的进展，为寻找其治疗靶点提供了新方向，针对不同靶点的药物在动物实验和临床试验中取得了一定的效果。本文将对ADPKD的发病机制及药物研究进展做一综述。

英文摘要：

Autosomal dominant polycystic kidney disease （ADPKD） is a comnlou inherited kidney disease, which is characterized by bilateral renal multiple fluid-filled cysts. ADPKD is the fourth cause of uremia, with a prevalence of about 1/1 000. Approximately half of the patients with ADPKD will progress to end-stage renal disease before the age of 60 years, hut there has been not any very effective therapies. In recent years, advances in the molecular mechanisms of this disease have provided new directions for seeking the therapeutic targets. Preclinieal models and clinical trials of the novel therapies have achieved positive results. This review focused on the progress of studies on pathogenesis and therapies of ADPKD.