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Therapeutic targets for polycystic kidney disease

机译：多囊肾的治疗靶标

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Introduction: 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.Areas covered: 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.Expert opinion: 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 -1-integrin receptor, as well as the intracellular integrator kinase, c-Src.
机译：简介：多囊肾病（PKD）是一种常见的遗传性疾病，其中肾肿大和功能丧失是由肾小管囊肿的逐渐扩张引起的。为了逆转PKD基因突变的不利影响并减缓囊性扩张，需要新药治疗。涵盖的领域：审查了导致PKD分子靶标鉴定的基础细胞生物学。具体的重点是在临床前早期阶段发表的研究。这些包括遗传和表观遗传调节剂，以及减缓囊性扩张和疾病进展的药物。特定药物和临床试验的讨论不在本文的范围之内。文献研究方法包括使用关键字组合的EndNote和PubMed在线搜索：多囊肾，临床前，分子靶标，信号转导，遗传调节剂，表观遗传学，治疗学，受体，激酶。在可能的情况下，参考与给定靶标有关的最新引文。专家意见：建议未来治疗开发中最有希望的靶标是靶向细胞膜上游信号传递事件的靶标，例如血管加压素2受体（AVPR2）。 ，EGFR / ErbB2和-1-整合素受体，以及细胞内整合子激酶c-Src。

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