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Gene Therapy Technique for Inherited Heart Problems in The Pipeline

According to the researchers at the University of Florida, Gainesville, mice based experiments were found to be successful in delivering genes to treat hereditary diseases of the heart.//

This new technique was found to be successful in mice affected with muscular dystrophy that damages the heart. 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 as having a genetic basis; all of the arrhythmias, problems that are due to a family of diseases called long Q-T syndrome, the heart failure category where many folks have been attempting to modify contractility with gen
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