A protein that targets the effects of a faulty gene could offer the first treatment targeting the major genetic cause of pulmonary arterial hypertension (PAH), according to research funded by the British Heart Foundation (BHF) and carried out at the University of Cambridge in the UK. Genetic evidence dating back to 2000, from research the BHF helped to fund, indicated that the absence or reduced activity of a particular protein, bone morphogenetic protein type II receptor (BMPR-II), leads to PAH. BMPR-II is important to the normal function of the blood vessels of the lungs. PAH is believed to affect approximately 6,500 people in the UK. This new study led by BHF Professor of Cardiopulmonary Medicine Dr. Nick Morrell and including expertise from Dr. Rajiv Machado at the University of Lincoln, UK, is the first to use a protein, called bone morphogenetic protein 9 (BMP9), to reverse the effects of reduced activity of BMPR-II and to reverse the PAH disease itself. The study was conducted in mice and rats, but also using cells from patients with PAH. It was published online on June 15, 2015 in Nature Medicine. The article is titled “Selective Enhancement of Endothelial BMPR-II with BMP9 Reverses Pulmonary Arterial Hypertension.” PAH is a chronic and debilitating disease that affects the blood vessels in the lungs, leading to heart failure, and leaving sufferers feeling breathless and exhausted. Current treatments only target the symptoms and prognosis remains poor. The only effective cure is a lung, or heart and lung, transplant, which has associated risks and complications. Once diagnosed with PAH, a person has a 30 per cent chance of dying within three years and the condition affects more women than men.
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