mTOR Inhibitors in Polycystic Kidney Disease

Autosomal dominant polycystic kidney disease (ADPKD) is a common cause of inherited renal failure1 that is characterized by the progressive formation of renal cysts, which leads to end-stage renal disease in mid-adulthood. Furthermore, massive renal enlargement has a number of untoward consequences, including chronic pain, hypertension, and cyst infections. Approximately 85% of ADPKD cases are caused by mutations in the PKD1 gene that encodes a large membrane receptor. The remaining 15% of cases result from mutations in the PKD2 gene, a calcium-permeable channel that binds to PKD1. The accelerated pace of scientific discovery in the field has led to an . . .