Clash to ‘cure’ the genetic heart condition that causes sudden death may be just a few years away | UK News
Just a few years from now, a “once-in-a-generation” stab could “cure” the inherited heart diseases that cause sudden death. scientists say.
An international team of researchers is developing the first cures for inherited cardiomyopathies by rewriting DNA with the aim of editing or blocking faulty genes.
They were awarded £30 million by the British Heart Foundation (BHF) to fund their research.
It is hoped that within the next few years, a “cure” injection could be given to patients like an injection into the arm, and could be used to prevent illness in patients. family members carrying the same faulty gene.
Hereditary cardiomyopathy that is precipitated by various cardiac abnormalities 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, which is very often caused by inherited cardiomyopathy, also known as hereditary cardiomyopathy.
All people with inherited cardiomyopathy have a 50-50 risk of passing the faulty genes on to each of their children, and often, several members of the same family develop heart failure, need a heart transplant, or die. when young.
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Professor Hugh Watkins, from the University of Oxford and principal investigator of the CureHeart project, said cardiomyopathy was “really common” and one in 250 people got it.
“There will be one or two schools at each school,” he told a news conference. “Every GP surgery will have some patients with these conditions, but there’s quite a bit of severity.”
He said not all patients would require the new therapy being proposed, but “a very large number” would benefit.
He added: “This is a once-in-a-generation opportunity to address families’ constant anxiety about sudden death, heart failure and the potential need for a heart transplant.
“After 30 years of research, we have discovered many genes and specific genetic errors that cause different cardiomyopathies and how they work. We believe we will have a gene therapy ready. to begin testing in clinical trials over the next 5 years.”
