Sirolimus attenuates disease progression in an orthologous mouse model of human autosomal dominant polycystic kidney disease

• Published in: Kidney international Vol. 78; no. 8; pp. 754 - 761
• Main Authors: Zafar, Iram, Ravichandran, Kameswaran, Belibi, Franck A., Doctor, R. Brian, Edelstein, Charles L.
• Format: Journal Article
• Language: English
• Published: Basingstoke Elsevier Inc 01.10.2010; Nature Publishing Group; Elsevier Limited
• Subjects: Animals; Biological and medical sciences; Body Weight - drug effects; Cysts; Disease Models, Animal; Disease Progression; Humans; Immunosuppressive Agents; Kidney - pathology; Kidney Function Tests; Kidneys; Malformations of the urinary system; Mechanistic Target of Rapamycin Complex 1; Medical sciences; Mice; Mice, Mutant Strains; Multiprotein Complexes; Nephrology. Urinary tract diseases; Organ Size - drug effects; polycystic kidney disease; Polycystic Kidney, Autosomal Dominant - drug therapy; Proteins - metabolism; signaling; Sirolimus - pharmacology; Sirolimus - therapeutic use; sirolimus polycystic kidney mouse; TOR Serine-Threonine Kinases; TRPP Cation Channels - genetics; polycystic kidney disease; signaling; sirolimus polycystic kidney mouse; Antineoplastic agent; Animal model; Nephrology; Prognosis; Sirolimus; Urology; Macrocycle; Signal transduction; Cyst; Evolution; Dominant character; Protein synthesis inhibitor; Benign neoplasm; Kidney disease; Human; Urinary system disease; Polycystic kidney; Rodentia; Lactone; Autosome; Macrolide; Genetic disease; Vertebrata; Antibiotic; Mammalia; Mouse; Animal; Autosomal character; Immunosuppressive agent; Antibacterial agent

In autosomal dominant polycystic kidney disease (ADPKD), abnormal proliferation of tubular cells drives cyst development and growth. Sirolimus, an inhibitor of the protein kinase mammalian target of rapamycin (mTOR) and a potent anti-proliferative agent, decreases cyst growth in several genetically...