ATS 2010, NEW ORLEANS— Gene therapy has been shown to have positive effects in rat models of pulmonary arterial hypertension (PAH), according to researchers at the University of Adelaide in Australia.
PAH is a life-threatening disease in which pressure in the blood vessels of the lungs increases, causing a back-pressure strain on the heart. In inherited forms of the disease, PAH is caused by a mutation in a receptor called bone morphogenetic protein receptor, type II (BMPR2). Even in some non-inherited forms of the disease, BMPR2 levels are low.