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Genetic Therapy Reverses Heart Disease in Mice

Trials in other animals have begun; humans would be up next

By Katherine Thompson,  Newser Staff

Posted Dec 1, 2008 11:52 AM CST

(Newser) – Damage to heart muscle can be stopped and maybe even reversed, but for now only in mice, the BBC reports. Blocking the activity of a specific type of genetic material that regulates gene expression, scientists found, avoided a type of cardiac scarring that leads to heart disease. "Heart function and tissue damage improved," a lead researcher told Reuters.

Researchers isolated a material known as microRNA-21, which was more common in failing hearts than in healthy ones. The results raised hopes that a possible treatment for humans may not be far off. The next step is testing in other animals, a process that has already begun. "This is one of the hottest topics in biology at the moment," said a researcher who called the study's results "exciting."

In laboratory mice, a chemical that blocks microRNA-21 prevented the scarring that usually leads to heart disease, and even reversed its effects in some cases.
In laboratory mice, a chemical that blocks microRNA-21 prevented the scarring that usually leads to heart disease, and even reversed its effects in some cases.   (Shutter Stock)
One type of microRNA, a piece of genetic material that regulates other gene activity, is largely responsible for releasing chemicals that lead to permanent scarring of the heart.
One type of microRNA, a piece of genetic material that regulates other gene activity, is largely responsible for releasing chemicals that lead to permanent scarring of the heart.   (Shutter Stock)
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There are already studies in large animals using micro-RNA inhibitors in heart disease - I can envisage that in a few years we will see this in human trials. - Eric Olson, profsesor at the University of Texas Southwestern Medical Center

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