Gene therapy stops hypertension in study with animals

Pulmonary arterial hypertension kills about half of those diagnosed within five years.

By Stephen Feller

NEW YORK, April 29 (UPI) -- Using a method of gene therapy, researchers slowed progression of pulmonary hypertension and improved arterial function in pigs, suggesting the deadly condition can be treated in humans, according to researchers.

The inhalation technique of delivering the genetic update is novel, say researchers at Mount Sinai who conceived it, as the success with pigs is the second test after the treatment was previously effective with rodents.


Pulmonary arterial hypertension, or PAH, occurs when blood pressure is too high from the heart to the lungs, affecting between 1 and 2 million people per year in the United States. There is no cure for the disease, which can lead to heart failure. It is twice as prevalent in women than men and kills about half of those diagnosed with it.

The high blood pressure is caused by changes in lung vessels that can lead to failure of the right ventricle of the heart and death, as well as thickening and narrowing of blood vessels resulting from a lack of sarcoplasmic reticulum calcium ATPase pump, or SERCA2a, which regulates calcium in vascular cells.

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"I'm excited that there is a potential new treatment for patients with this deadly disease," Dr. Roger Hajjar, director of the Cardiovascular Research Center at Mount Sinai Hospital, said in a press release. "By tailoring the gene therapy, it looks like we can halt the proliferation of smooth muscle cells in the blood vessels. This should help restore function and improve survival in human patients."


For the study, published in the Journal of the American College of Cardiology, researchers treated 20 Yorkshire swine bred to have post-capillary PAH, giving 10 an aerosolized viral vector carrying the SERCA2a gene and the other 10 saline.

In tests two months after the treatment, researchers found the therapeutic genes were present and functioning, and that heart and lung function had improved in pigs receiving the SERCA2a.

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"There is considerable need for new treatments that improve functional capacity, symptoms and survival," Hajjar said. "This treatment with gene therapy offers a novel method of treating a deadly disease that disproportionately affects young adults and women."

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