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Jefferson scientists use gene therapy to restore function of damaged heart cells in laboratory

-stage heart failure with an adenovirus that encoded both ARKct a peptide that can block ARK 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 then were able to use a video camera to actually measure how strong the individual heart cells were beating.

"We put the ARKct into the cells, and failing human hearts become more like normal hearts, based on their ability to contract," Dr. Koch says. "This is the first work in actual human cells to show efficacy of ARKct and the enzyme ARK1 as targets for heart failure.

"This study is the last proof of concept," he adds, noting that years of previous work in various animal models enabled the research team to reach this point. "Now we are dealing with human cells from failing human hearts."

Further animal studies are planned, which, he notes, should lead to eventual human clinical trials.


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Contact: Steve Benowitz
steven.benowitz@jefferson.edu
215-955-5291
Thomas Jefferson University
13-Nov-2003


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