Researchers are confident that they will soon be able to cure inherited heart muscle diseases, a leading cause of sudden death in young people.
The British Heart Foundation has pledged £30 million to the scientists behind the CureHeart trial, who believe their gene therapy technology could silence or correct the faulty genes linked to cardiomyopathies.
Cardiomyopathy is a disease which leaves heart muscle weakened or distorted, and functionally impaired. This can cause breathlessness, palpitations, chest pain or blackouts, and frequently leads to fatal heart failure.
Although its symptoms can be managed by surgery, medication or devices such as pacemakers, heart failure has no cure.
Now that the specific genetic faults responsible for various cardiomyopathies have been uncovered, the CureHeart research team believes that their gene therapy could be ready for testing in human clinical trials in just five years, following previous research in human and animal cells.
Their treatment, which would be administered through a simple injection to the arm, could halt heart damage in those already living with genetic cardiomyopathies and prevent the disease from developing in family members who carry a faulty gene.
Prof Sir Nilesh Samani, medical director at the British Heart Foundation, described the team’s work as a defining moment for cardiovascular disease, emphasising that their breakthrough will offer hope to thousands of families affected by genetic heart conditions.
“Not only could CureHeart be the creators of the first cure for inherited heart muscle diseases by tackling killer genes that run through family trees, it could also usher in a new era of precision cardiology.
“Once successful, the same gene editing innovations could be used to treat a whole range of common heart conditions where genetic faults play a major role.”
It is estimated that one in 250 people worldwide are affected by inherited heart muscle diseases, with a 50:50 risk they will pass faulty genes on to their children.
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