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RGLS4326


Autosomal Dominant Polycystic Kidney Disease (ADPKD)

ADPKD, caused by the mutations of PKD1 or PKD2, is among the most common monogenetic disorders and a leading genetic cause of end-stage renal disease. The clinical hallmark of this disease is the presence of numerous renal tubule-derived cysts.  Excessive proliferation, a central pathological feature, fuels the relentless expansion of cysts, ultimately causing end-stage renal disease. ADPKD affects approximately 600,000 people in the US and 12.5 million people worldwide.