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Trying gene therapy to create biological pacemaker

WASHINGTON — Scientists are creating a biological pacemaker by injecting a gene into the hearts of sick pigs that changed ordinary cardiac cells into a special kind that induces a steady heartbeat.

The study, published Wednesday, is one step toward developing an alternative to electronic pacemakers that are implanted into 300,000 Americans a year.

"There are people who desperately need a pacemaker but can't get one safely," said Dr. Eduardo Marban, director of the Cedars-Sinai Heart Institute in Los Angeles, who led the work. "This development heralds a new era of gene therapy" that one day might offer them an option.

Your heartbeat depends on a natural pacemaker, a small cluster of cells — it's about the size of a peppercorn, Marban says — that generates electrical activity. Called the sinoatrial node, it acts like a metronome to keep the heart pulsing at 60 to 100 beats a minute or so, more when you're active. If that node quits working correctly, hooking the heart to an electronic pacemaker works very well for most people.

But about 2 percent of recipients develop an infection that requires the pacemaker to be removed for weeks until antibiotics wipe out the germs, Marban said. And some fetuses are at risk of stillbirth when their heartbeat falters, a condition called congenital heart block.

For more than a decade, teams of researchers have worked to create a biological alternative that might help those kinds of patients.

Marban's newest attempt uses gene therapy to reprogram a small number of existing heart muscle cells so they start looking and acting like natural pacemaker cells instead.

Because pigs' hearts are so similar to human hearts, Marban's team studied the approach in 12 laboratory pigs with a defective heart rhythm.

Two days after treatment, pigs had faster heartbeats than control pigs who didn't receive the gene, the researchers reported in the journal Science Translational Medicine. That heart rate automatically fluctuated, faster during the day. The treated animals also became more active, without signs of side effects.

"In essence, we created a new sinoatrial node," Marban said. "The newly created node then takes over as a functional pacemaker, bypassing the need for implanted electronics and hardware."

Marban said longer-term animal studies are under way, and he hopes to begin first-step human studies in about three years.

Dr. Eduardo Marban, chief of the Cedars-Sinai Heart Institute in Los Angeles, led the pacemaker study. Traditional pacemakers are implanted into 300,000 Americans a year.

Associated Press

Dr. Eduardo Marban, chief of the Cedars-Sinai Heart Institute in Los Angeles, led the pacemaker study. Traditional pacemakers are implanted into 300,000 Americans a year.

Trying gene therapy to create biological pacemaker 07/16/14 [Last modified: Wednesday, July 16, 2014 10:20pm]

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