Treatment with RGLS4326 suppresses cyst growth in human ADPKD models in vitro, and slows cyst growth in Pkd2 conditional knockout mice (Pkd2flox/flox:Pkhd1-Cre) as well as Pcy/CD1 and Pcy/DBA mice which develop polycystic kidney disease with mutations in NPHP3 (nephronophthisis 3) and are used as mouse models for long-term treatment [61]. The gene discussed is PKHD1; the disease is polycystic kidney disease.