Our lab focuses on understanding the pathomechanisms of polycystic kidney disease (PKD) and determining potential novel therapies for PKD treatment.
What is Polycystic Kidney Disease?
Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disorder and a potentially life-threatening condition. It is characterized by the progressive development of fluid-filled cysts, chronic renal inflammation, and fibrosis, ultimately leading to kidney failure.ADPKD affects approximately 1 in every 400 to 1,000 individuals, with over 600,000 people in the United States and more than 12 million people affected worldwide. The disease is primarily caused by mutations in the PKD1 gene (about 85% of cases) or the PKD2 gene (15%), which encode the proteins polycystin-1 (PC-1) and polycystin-2 (PC-2), respectively.Most patients with ADPKD develop progressive kidney dysfunction in midlife, and the disease accounts for nearly 9% of individuals requiring dialysis or kidney transplantation. Despite its prevalence and severity, treatment options remain limited. Currently, tolvaptan, a vasopressin V2 receptor antagonist, is the only FDA-approved therapy for ADPKD.
Our Research:
We are focused on understanding the underlying mechanisms of ADPKD and developing new therapeutic strategies. Specifically, we aim to:
Investigate whether promoting the resolution of renal inflammation can reduce cyst growth, kidney fibrosis, and loss of kidney function.
Explore the structure-function relationship of PC-1, the protein product of PKD1, which is mutated in the 85% of ADPKD cases.