Sept. 28, 2001 Gene therapy corrects heart failure in rat model
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September 28, 2001

Gene therapy corrects heart failure in rat model

An MGH research group, which previously identified an important component leading to heart failure, has successfully fixed the problem in a rat model of the disease. The results are published in the Sept. 18 issue of Circulation.

"Fixing this one problem corrects a large number of abnormalities in the heart," say principal authors Federica del Monte MD, PhD, and Roger Hajjar, MD, of the MGH Cardiovascular Research Center. The MGH researchers have been studying a molecular pump that regulates calcium flow and allows the heart to contract and relax. "Heart failure in the aging is due to the fact that this pump is abnormal. It doesn't allow the heart to relax and fill appropriately," says Hajjar.

Del Monte and Hajjar's team used a model system, in which laboratory rats develop symptoms similar to those of humans with heart failure. The researchers discovered that fixing the calcium pump through gene replacement significantly improved the rate of survival.092801hajjar.jpg (22421 bytes)

"We found that four weeks after we treated these rats with the gene encoding the calcium pump, survival was 63 percent compared with 9 percent for rats that didn't receive the gene," says Hajjar (right).

The researchers say that they hope their work eventually will lead to new methods of treating heart failure. MGH co-authors of the study are: Eric Williams; Djemal Lebeche, PhD; Ulrich Schmidt, MD, PhD; and Anthony Rosenzweig, MD.


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