Department of Biomedical Science, Faculty of Medicine, University of Malaya, 50603, Kuala Lumpur, Malaysia.
Renal Division, Department of Medicine, University of Malaya, 50603, Kuala Lumpur, Malaysia.
Clin Exp Nephrol. 2021 Nov;25(11):1163-1172. doi: 10.1007/s10157-021-02111-x. Epub 2021 Jul 12.
Autosomal dominant polycystic kidney disease (ADPKD) is a monogenic kidney disorder that impairs renal functions progressively leading to kidney failure. The disease affects between 1:400 and 1:1000 ratio of the people worldwide. It is caused by the mutated PKD1 and PKD2 genes which encode for the defective polycystins. Polycystins mimic the receptor protein or protein channel and mediate aberrant cell signaling that causes cystic development in the renal parenchyma. The cystic development is driven by the increased cyclic AMP stimulating fluid secretion and infinite cell growth. In recent years, natural product-derived small molecules or drugs targeting specific signaling pathways have caught attention in the drug discovery discipline. The advantages of natural products over synthetic drugs enthusiast researchers to utilize the medicinal benefits in various diseases including ADPKD.
Overall, this review discusses some of the previously studied and reported natural products and their mechanisms of action which may potentially be redirected into ADPKD.
常染色体显性多囊肾病（ADPKD）是一种单基因肾脏疾病，会逐渐损害肾功能，导致肾衰竭。这种疾病在全球范围内的发病率为 1:400 至 1:1000。它是由 PKD1 和 PKD2 基因突变引起的，这些突变基因编码缺陷型多囊蛋白。多囊蛋白模拟受体蛋白或蛋白通道，并介导异常的细胞信号转导，导致肾脏实质中的囊性发育。囊性发育是由增加的环 AMP 刺激液分泌和无限的细胞生长驱动的。近年来，天然产物衍生的小分子或针对特定信号通路的药物在药物发现领域引起了关注。天然产物相对于合成药物的优势促使研究人员在包括 ADPKD 在内的各种疾病中利用其药用价值。
总的来说，本综述讨论了一些先前研究和报道的天然产物及其可能潜在用于治疗 ADPKD 的作用机制。
