In vitro analysis of patient-derived loss-of-function KCNK3 variants showed successful recovery of channel function in response to a phospholipase inhibitor, suggesting a novel therapeutic avenue for PAH.1 2 Similarly, experimental therapies targeting the polyamine transport system in cancer23 can now be tested in ATP13A3-targeted model systems relevant to PAH. The gene discussed is ATP13A3; the disease is pulmonary arterial hypertension.