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Inhalable gene therapy may aid PAH patients

Authored by James Sheppard

US researchers have shown in a new study that gene therapy administered through a nebulizer-like inhalation device can completely reverse pulmonary arterial hypertension (PAH) in rat models of the disease. The research was conducted at The Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai. The research was published in the most recent version of the journal Circulation.

Roger Hajjar. MD, Director of Cardiovascular Research Center said ‘The gene therapy could be delivered very easily to patients through simple inhalation – just like the way nebulizers work to treat asthma. We are excited about testing this therapy in PAH patients who are in critical need of intervention.’

In the lab, researchers also found in the pulmonary artery of PAH patient tissue samples reduced expression of SERCA2a, an enzyme critical for proper pumping of calcium in calcium compartments within cells. SERCA2a gene therapy could be sought as a promising therapeutics intervention in PAH.  The same SERCA2a dysfunction also occurs in heart failure. This new study utilizes the same gene therapy currently being tested in patients to reverse congestive heart failure in a large phase III study being conducted in Europe and the US

Dr Hajjar went on to say, ‘what we have shown is that gene therapy restores function of this crucial enzyme in diseased lungs. We are delighted with these new findings because it suggests that a gene therapy is already showing great benefit in congestive heart failure patients and may be able to help PAH patients who currently have no good treatment options and are in critical need of a life sustaining therapy. We are now on a path towards clinical trials in the near future.’

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