Department of Biological Science, Sookmyung Women's University, Seoul 140-742, Korea.
BMB Rep. 2011 Jun;44(6):359-68. doi: 10.5483/BMBRep.2011.44.6.359.
Polycystic kidney disease (PKD) is a common genetic disorder in which extensive epithelial-lined cysts develop in the kidneys. In previous studies, abnormalities of polycystin protein and its interacting proteins, as well as primary cilia, have been suggested to play critical roles in the development of renal cysts. However, although several therapeutic targets for PKD have been suggested, no early diagnosis or effective treatments are currently available. Current developments are active for treatment of PKD including inhibitors or antagonists of PPAR-γ, TNF-α, CDK and VEGF. These drugs are potential therapeutic targets in PKD, and need to be determined about pathological functions in human PKD. It has recently been reported that the alteration of epigenetic regulation, as well as gene mutations, may affect the pathogenesis of PKD. In this review, we will discuss recent approaches to PKD therapy. It provides important information regarding potential targets for PKD.
多囊肾病(PKD)是一种常见的遗传性疾病,其特征为肾脏广泛发生内衬上皮的囊肿。在以前的研究中,多囊蛋白及其相互作用蛋白以及初级纤毛的异常被认为在肾囊肿的发展中起关键作用。然而,尽管已经提出了几种 PKD 的治疗靶点,但目前尚无早期诊断或有效治疗方法。目前正在积极开发治疗 PKD 的方法,包括 PPAR-γ、TNF-α、CDK 和 VEGF 的抑制剂或拮抗剂。这些药物是 PKD 的潜在治疗靶点,需要确定它们在人类 PKD 中的病理功能。最近有报道称,表观遗传调控的改变以及基因突变可能影响 PKD 的发病机制。在这篇综述中,我们将讨论 PKD 治疗的最新方法。它为 PKD 的潜在靶点提供了重要信息。