"First do no harm": kidney drug targeting to avoid toxicity in ADPKD

Autosomal dominant polycystic kidney disease (ADPKD) is the most commonly inherited kidney disease, affecting 12 million individuals worldwide and caused by the mutation of one of two genes, Pkd1 or Pkd2. The disease is characterized by overproliferation of renal epithelial cells and gradual enlargement of fluid-filled cysts, leading to displacement and destruction of adjacent renal parenchyma and ultimately the loss of kidney function. Clinical presentation of ADPKD typically occurs by the age of 30, and by the age of 60, most patients will require dialysis or a kidney transplant.