Drug discovery for polycystic kidney disease

• Published in: Acta pharmacologica Sinica Vol. 32; no. 6; pp. 805 - 816
• Main Authors: Sun, Ying, Zhou, Hong, Yang, Bao-xue
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.06.2011; Nature Publishing Group
• Subjects: Animals; Apoptosis - drug effects; Biomedical and Life Sciences; Biomedicine; Cell Proliferation - drug effects; Clinical Trials as Topic; Disease Models, Animal; Drug Discovery; Drugs, Investigational - administration & dosage; Drugs, Investigational - pharmacology; Drugs, Investigational - therapeutic use; Epithelium - drug effects; Epithelium - metabolism; Epithelium - pathology; Humans; Immunology; Internal Medicine; Medical Microbiology; Pharmacology/Toxicology; PKD; Polycystic Kidney Diseases - drug therapy; Polycystic Kidney Diseases - etiology; Polycystic Kidney Diseases - metabolism; Polycystic Kidney Diseases - pathology; Review; TRPP Cation Channels - genetics; TRPP Cation Channels - metabolism; TRPP Cation Channels - physiology; Vaccine; 发病机制; 肾功能衰竭; 肾病; 药物发现; 药物治疗; 药物靶标; 遗传性疾病; kidney; candidate drugs; drug discovery; animal model; polycystic kidney disease
• ISSN: 1671-4083; 1745-7254; 1745-7254
• DOI: 10.1038/aps.2011.29

In polycystic kidney disease （PKD）, a most common human genetic diseases, fluid-filled cysts displace normal renal tubules and cause end-stage renal failure. PKD is a serious and costly disorder. There is no available therapy that prevents or slows down the cystogenesis and cyst expansion in PKD. Numerous efforts have been made to find drug targets and the candidate drugs to treat PKD. Recent studies have defined the mechanisms underlying PKD and new therapies directed toward them. In this review article, we summarize the pathogenesis of PKD, possible drug targets, available PKD models for screening and evaluating new drugs as well as candidate drugs that are being developed.