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Researchers at hospitals in London and Glasgow plan to use an engineered virus to smuggle DNA into the heart in the first ever clinical trial of 'gene therapy' for heart failure.

The technique, which has been 20 years in development, could help the 750,000 people in the UK living with heart failure.

The condition results from damage to the heart, typically left over from a heart attack or side-effects of powerful chemotherapy drugs. After the heart is starved of oxygen, cells die and the remaining heart cells become fatigued.

“Once heart failure starts, it progresses into a vicious cycle where the pumping becomes weaker and weaker, as each heart cell simply cannot respond to the increased demand.” said Dr Alex Lyon, a cardiologist at  Royal Brompton Hospital who is leading the trial in the UK.

“Our goal is to fight back against heart failure by targeting and reversing some of the critical molecular changes arising in the heart when it fails,” he said.

People with heart failure can find it difficult to walk long distances or climb stairs. The disease is usually progressive, with heart function gradually weakening over time. The only solutions are heart transplant or surgically implanted pumps - known as a ventricular assist device - to maintain blood flow.

The gene therapy adds a repair gene to failing heart cells which produces more of a protein called SERCA2a which regulates the availability of calcium in the heart. Without ample supplies of calcium, heart muscles are unable to contract properly or relax properly between contractions – two key symptoms of heart failure.

Early tests of the technique show it appears to be safe, now 200 patents with severe heart failure are being recruited in Europe and America to show that it is effective. Doctors in the UK want to recruit 10 or more patients into the trial here.

Not all heart failure patients may benefit, however. The virus used to insert the DNA is so common many patients may have antibodies to it, meaning their immune systems will destroy the virus before it can infect the heart cells.


Read the full story:

Gene therapy hope for heart failure patients - Channel 4