Molecular pathways and therapies in autosomal-dominant polycystic kidney disease.
Autosomal-dominant polycystic kidney disease (ADPKD) is the most prevalent inherited renal disease, characterized by multiple cysts that can eventually lead to kidney failure. Studies investigating the role of primary cilia and polycystins have significantly advanced our understanding of the pathogenesis of PKD. This review will present clinical and basic aspects of ADPKD, review current concepts of PKD pathogenesis, evaluate potential therapeutic targets, and highlight challenges for future clinical studies.
Department of Medicine, Division of Nephrology, Medical University of South Carolina, Charleston, South Carolina; and Ralph Johnson VA Medical Center, Charleston, South Carolina.
Takamitsu
T
Saigusa
Department of Medicine, Division of Nephrology, Medical University of South Carolina, Charleston, South Carolina; and Ralph Johnson VA Medical Center, Charleston, South Carolina bellpd@musc.edu.
P Darwin
PD
Bell
D000818:Animals; D002923:Cilia; D020022:Genetic Predisposition to Disease; D005820:Genetic Testing; D006801:Humans; D007668:Kidney / Q000187:drug effects / Q000378:metabolism / Q000473:pathology; D058990:Molecular Targeted Therapy; D009154:Mutation; D010641:Phenotype; D016891:Polycystic Kidney, Autosomal Dominant / Q000175:diagnosis / Q000453:epidemiology / Q000235:genetics* / Q000378:metabolism / Q000628:therapy*; D011237:Predictive Value of Tests; D012307:Risk Factors; D015398:Signal Transduction; D050396:TRPP Cation Channels / Q000235:genetics*; D016896:Treatment Outcome
D050396:TRPP Cation Channels