autologous mononuclear cells expressing human nitric oxide synthase

Autologous mononuclear cells expressing human nitric oxide synthase (heNOS) is an experimental cell-based gene therapy developed by Northern Therapeutics for the treatment of idiopathic pulmonary arterial hypertension (iPAH). The therapy involves harvesting a patient's own mononuclear cells, genetically engineering them ex vivo to express the human endothelial nitric oxide synthase (eNOS) gene, and then reinfusing them into the pulmonary circulation. The engineered cells produce nitric oxide, a potent vasodilator, which helps to restore nitric oxide bioavailability, reduce pulmonary vascular resistance, and improve pulmonary hemodynamics. This approach was evaluated in the Phase I PHACT (Pulmonary Hypertension: Assessment of Cell Therapy) clinical trial.

Brand names

Aurora-GT

Other names

autologous mononuclear cells expressing heNOSheNOS-transfected mononuclear cellsPHACT cell therapy