Gene Therapy Can Reverse Pulmonary Hypertension, Study Suggests


A Heart and Stroke Foundation researcher has discovered what could be the first truly effective breakthrough in the diagnosis and treatment of pulmonary hypertension, a devastating, life-threatening condition which results in an enlargement of the heart.

"We have discovered an early warning system in a protein called PIM-1," Dr. Sébastien Bonnet told the Canadian Cardiovascular Congress 2010, co-hosted by the Heart and Stroke Foundation and the Canadian Cardiovascular Society.

Dr. Bonnet has established that the PIM-1 cells can be used as markers of pulmonary hypertension.

"Blood samples were taken from patients to measure PIM-1 expression in the blood," says Dr. Bonnet, who is a professor at Laval University and a researcher at Centre hospitalier universitaire de Québec. "We were surprised to find that the greater the PIM-1 levels, the more severe the pulmonary hypertension in the patient."

He says this opens the doors to using regular blood tests to look at PIM-1 levels. "If there is a slight increase in PIM-1, we will know that something is going on." This is important since the condition is under-diagnosed and often not discovered until it is in a late stage. Without earlier treatment it has a very poor prognosis. The condition has traditionally been diagnosed by a six minute walking test.

PIM-1 also offers the opportunity to move beyond the diagnosis of pulmonary hypertension to treatment. By blocking the PIM-1 protein, researchers were able to reverse the condition.

"This is a remarkable finding," says Dr. Bonnet. "We have found that using gene therapy to inhibit the inappropriate activation of this protein is a novel and effective therapy that can reverse the disease altogether."

(ScienceDaily)

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