Jab to ‘cure’ genetic heart disease that causes sudden death could be just a few years away | UK News

A ‘one-off’ vaccine that could ‘cure’ the genetic heart disease that causes sudden death is only a few years away. scientists say.

An international team of researchers is developing the first cures for inherited heart muscle disease by rewriting DNA to edit or switch off faulty genes.

They have been awarded £30million by the British Heart Foundation (BHF) to fund their research.

The hope is that within a few years, patients can be given a “curative” injection as a stab in the arm and used to prevent disease in family members who carry the same faulty gene.

Hereditary myocardial disease is caused by various abnormalities in the heart, but can cause sudden death or progressive heart failure.

Every week in the UK, 12 people under the age of 35 die from undiagnosed heart disease, very often caused by an inherited heart muscle disease also known as genetic cardiomyopathy.

All people with genetic cardiomyopathies have a 50:50 risk of passing faulty genes to their children, and often several members of the same family will develop heart failure, require a heart transplant, or die at a young age.

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Professor Hugh Watkins of the University of Oxford and lead researcher on the CureHeart project said cardiomyopathies are “really common”, affecting one in 250 people.

“There will be one or two in every school,” he said at a briefing. “Any GP practice will have multiple patients with these conditions, but there is a range of severities.”

He said not all patients would need the proposed new therapy, but “a very large number” would benefit.

He added: “This is our unique opportunity to free families from the constant worry of sudden death, heart failure and the possible need for a heart transplant.

“After 30 years of research, we have discovered many of the genes and specific genetic errors responsible for various cardiomyopathies and how they work. We believe we will have a gene therapy that is ready to be tested in clinical trials within the next five years. “