University of Colorado Denver, Division of Renal Diseases and Hypertension, Box C281, 12700 East 19th Ave, Aurora, CO 80045, USA.
Expert Opin Investig Drugs. 2010 Mar;19(3):315-28. doi: 10.1517/13543781003588491.
Autosomal dominant (AD) polycystic kidney disease (PKD) is the most common life-threatening hereditary disorder. There is currently no therapy that slows or prevents cyst formation and kidney enlargement in humans. An increasing number of animal studies have advanced our understanding of molecular and cellular targets of PKD.
The purpose of this review is to summarize the molecular and cellular targets involved in cystogenesis and to update on the promising therapies that are being developed and tested based on knowledge of these molecular and cellular targets.
Insight into the pathogenesis of PKD and how a better understanding of the pathogenesis of PKD has led to the development of potential therapies to inhibit cyst formation and/or growth and improve kidney function.
The results of animal studies in PKD have led to the development of clinical trials testing potential new therapies to reduce cyst formation and/or growth. A vasopressin V2 receptor antagonist, mTOR inhibitors, blockade of the renin-angiotensin system and statins that reduce cyst formation and improve renal function in animal models of PKD are being tested in interventional studies in humans.
常染色体显性遗传（AD）多囊肾病（PKD）是最常见的危及生命的遗传性疾病。目前尚无任何疗法可以减缓或阻止人类的囊肿形成和肾脏增大。越来越多的动物研究提高了我们对 PKD 的分子和细胞靶点的认识。
本综述的目的是总结参与囊肿发生的分子和细胞靶点，并根据这些分子和细胞靶点的知识，更新正在开发和测试的有前途的疗法。
深入了解 PKD 的发病机制，以及对 PKD 发病机制的更好理解如何导致抑制囊肿形成和/或生长以及改善肾功能的潜在疗法的发展。
PKD 的动物研究结果导致了临床试验的发展，以测试减少囊肿形成和/或生长的潜在新疗法。加压素 V2 受体拮抗剂、mTOR 抑制剂、阻断肾素-血管紧张素系统和他汀类药物可减少 PKD 动物模型中的囊肿形成并改善肾功能，目前正在对人类进行干预研究。
