Wilson Patricia D
a University College of London, Royal Free Hospital, UCL Centre for Nephrology , Rowland Hill Street, London, NW3 2PF, UK.
Expert Opin Ther Targets. 2016;20(1):35-45. doi: 10.1517/14728222.2015.1083979. Epub 2015 Aug 31.
Polycystic kidney disease (PKD) is a common genetic disease in which renal enlargement and loss of function is caused by progressive expansion of tubular cysts. To reverse the detrimental effects of PKD gene mutation(s) and to slow cystic expansion, new drug therapies are required.
The underlying cell biology leading to identification of molecular targets for PKD is reviewed. Specific focus is on studies published at the early pre-clinical level. These include genetic and epigenetic modulators, and drugs to slow cystic expansion and disease progression. Discussion of specific drugs and clinical trials is not within the scope of this article. Literature research methods included EndNote and PubMed online searches using keyword combinations: polycystic kidneys disease, pre-clinical, molecular targets, signal transduction, genetic modulators, epigenetic, therapeutic, receptors, kinases. Where possible, the most recent citations concerning a given target are referenced.
It is suggested that the most promising targets for future therapeutic development are those that target upstream signaling events at cell membranes, such as the vasopressin-2 receptor (AVPR2), EGFR/ErbB2, and the β-1-integrin receptor, as well as the intracellular integrator kinase, c-Src.
多囊肾病（PKD）是一种常见的遗传性疾病，肾小管囊肿的逐渐扩张会导致肾脏肿大和功能丧失。为了逆转PKD基因突变的有害影响并减缓囊肿扩张，需要新的药物治疗方法。
本文综述了导致PKD分子靶点确定的基础细胞生物学。特别关注临床前早期发表的研究。这些研究包括基因和表观遗传调节剂，以及减缓囊肿扩张和疾病进展的药物。本文不讨论具体药物和临床试验。文献研究方法包括使用关键词组合“多囊肾病、临床前、分子靶点、信号转导、基因调节剂、表观遗传、治疗、受体、激酶”在EndNote和PubMed在线搜索。在可能的情况下，引用了有关给定靶点的最新文献。
建议未来治疗发展最有前景的靶点是那些针对细胞膜上游信号事件的靶点，如血管加压素2受体（AVPR2）、表皮生长因子受体/ErbB2和β-1整合素受体，以及细胞内整合激酶c-Src。
