One-time-only gene therapy could take care of atherosclerosis for life

Public Enemy No. 1 for cardiovascular specialists is plaque. In atherosclerosis, they are fatty lesions that form on the inner lining of blood vessels, narrowing arteries, restricting blood flow and causing chest pains in patients. If the plaques rupture, resulting blood clots can cause heart attacks or strokes. Right now, the condition is treated with cholesterol-lowering statin drugs, but not everybody can tolerate statins and they must be taken daily for life--with no guarantee that they will prevent heart attack and stroke. Now, a researcher at the University of Washington has devised a one-time-only method of delivering gene therapy into an arterial wall, protecting it from developing atherosclerosis despite ongoing high blood cholesterol.

"Introducing into the blood vessel wall genes that protect against atherosclerosis is potentially an effective means of preventing or reversing plaque formation and inflammation," UW researcher David Dichek said in a release. "As applied in our study, the introduced genes can produce proteins that counteract the fundamental processes that drive atherosclerosis, including preventing lipid accumulation inside the artery wall and decreasing recruitment of inflammatory cells. We found both of these effects."

The researchers pointed out that the therapeutic gene would be delivered directly to the site of atherosclerosis development: the blood vessel wall, minimizing side effects for healthy cells and maximizing the effect of the drug.

The technique seemed to work in rabbits, anyway, and Dichek says he is hopeful that it will eventually eliminate the lifelong threat of atherosclerosis in humans, too.

- read the release
- and the abstract in Molecular Therapy

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