Pathogenesis and potential therapy of autosomal dominant polycystic kidney disease


  • O.O. Melnyk Specialized Medical Center “Optima-Pharm”, Kyiv, Ukraine



autosomal dominant polycystic kidney disease, polycystin-1, polycystin-2, cilia, treatment


Autosomal dominant polycystic kidney disease (ADPKD) is a hereditary disease characterized by progressive growth of the cyst and an increase in the total volume of the kidneys which leads to kidney failure. The main causes of ADPKD are mutations in the genes PKD1 and PKD2 which encode the formation of polycystin-1 and polycystin-2 proteins. There is a connection between structural and functional defects in the primary cilia with the ADPKD. The most promising drugs for the treatment of ADPKD today are vasopressin-2 receptor antagonists, m-TOR and c-AMP inhibitors.


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