MONDAY, July 31 (HealthDay News) -- A new method of gene therapy for hereditary heart conditions proved successful in animal tests and holds promise for long-lasting treatments in humans, say University of Florida researchers.
The approach delivers corrective genes with a single injection into the veins. It successfully reversed symptoms in mice with a form of muscular dystrophy that damages the heart.
The researchers also tested this virus-based delivery system in monkeys. The genes were readily absorbed by the monkeys' hearts, and the effect persisted for months.
The findings were published in the July 27 online edition of the journal Circulation Research.
Studies of this gene therapy method in people with Pompe disease -- a rare form of muscular dystrophy that's usually fatal within the first year of life -- could begin as early as next year.
"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," senior author and pediatric cardiologist Dr. Barry J. Byrne said in a prepared statement.
"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," Byrne said.
More information
The U.S. National Library of Medicine has more about gene therapy.
