Gene Therapy Restores Damaged Heart Cells in Lab

Their work was reported in the April 6, 2004, issue of Circulation.

For some time, researchers have known that the beta-adrenergic receptor system fails to work properly in patients with congestive heart failure. So the current investigators used a virus to carry a gene into the heart cells of congestive heart failure patients that blocks the activity of beta-adrenergic kinase (ßark1), which is increased in failing hearts and contributes to the loss of the heart's contractile strength. When the activity of this enzyme was blocked, the heart cells were able to contract at normal strength and their overall performance improved.

The researchers infected the heart cells from patients who underwent cardiac transplantation with an adenovirus that encoded both ßarkct, a peptide that can block ßark, and a so-called reporter gene protein, which glows green. The latter provided a signal to the scientists that the inhibitor was indeed present in the heart cells. They were then able to use a video camera to actually measure how strong the individual heart cells were beating.

"This is the first work in actual human hearts to show efficacy of ßarkct as a potential therapy and more importantly, proves that the enzyme ßark1 is a target for heart failure treatment,” said Walter Koch, Ph.D., director of the Center for Translational Medicine of the Department of Medicine at Jefferson Medical College (Philadelphia, PA, USA; www.jefferson.edu/jmc), who conducted the study along with colleagues from Duke University Medical Center (Durham, NC, USA; www.mc.duke.edu).





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