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UF scientists test improved gene therapy method for hereditary heart conditions

A new way of delivering corrective genes with a single injection into a vein holds promise for long-lasting treatments of hereditary diseases of the heart, University of Florida researchers report.

UF researchers used the approach to successfully reverse symptoms in mice with a form of muscular dystrophy that damages the heart. They also tested the virus-based delivery method in monkeys and found genes were readily absorbed by heart muscle cells, and the effect persisted for months.

The findings, published July 27 in the online edition of Circulation Research, pave the way for studies in humans that could begin as soon as early next year for patients with Pompe disease, a rare form of muscular dystrophy that is usually fatal in the first year of life.

"Nine years ago we knew we could get long-term gene expression in the heart but it was with direct injection into the heart muscle and it was inefficient," said UF pediatric cardiologist Barry J. Byrne, M.D., Ph.D., the paper's senior author and director of the Powell Gene Therapy Center. "The difference here is that we can deliver a much lower dose of the vector into a vein like any other drug, and the corrective gene collects in the heart."

Scientists say gene therapy looks increasingly feasible for the treatment of cardiovascular conditions linked to faulty genes or congenital metabolic diseases, including atherosclerosis, stroke, muscular dystrophy and an enlargement of the heart muscle known as dilated cardiomyopathy.

But efforts to begin testing it in people have been slowed by the need to find ways to deliver corrective genes easily and efficiently, so they go where they are needed. A number of conditions, for example, affect both heart and skeletal muscle and will require the widespread delivery of genes throughout the body, instead of to a localized site, to prevent or correct disease.

"There are many forms of adult heart disease that are now well-understood
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Source:University of Florida


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