First gene therapy to calm pigs' out-of-sync hearts

"The number one killer in developed countries is cardiac death, generally sparked by an event that leads to arrhythmia."

Scientists at Johns Hopkins have developed a gene therapy that, within a week, quells abnormal rhythms in pig hearts, the animal hearts most similar to human. It's believed to be the first use of gene therapy for cardiac arrhythmias, the researchers say, and one with "a strong possibility" of transfer to human heart disease.

"We've effectively treated an arrythmia in a well-tested animal model, using genes delivered by routine catheter methods no open chest, no contrivances, just simple modifications of existing technology," says Eduardo Marban, M.D., Ph.D., one of the scientists.

"This is the first proof-of-concept work," says Marban, "and the first clear step toward a clinically useful approach."

A report of the research appears in the December issue of the journal Nature Medicine.

Arrhythmias arise from two sources, the researchers say. One is a basic structural abnormality in the heart's pacemakers. The other develops after a heart attack or other insult to the heart. "Shortly after such an event," says team leader Kevin Donahue, M.D., "cellular changes, such as scarring, take place in the heart and make tissue more prone to arrhythmias. The number one killer in developed countries is cardiac death, generally sparked by an event that leads to arrhythmia."

Approximately one in every hundred people in this country suffers from arrythmias of one sort or another, according to the American Heart Association. While some heart rhythm disturbances are relatively benign, others, by disrupting the heart's coordinated pumping, are an immediate cause of collapse and death.

"Drug treatments can help, but don't fix the underlying causes of the problem," Donahue adds, "and continued use can actually encourage arrhythmias in some people. Pacemakers or defibrillators are expensive and

Contact: Marjorie Centofanti
Johns Hopkins Medical Institutions

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