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Scientists to rewrite DNA in search for heart disease cure

The British Heart Foundation’s ‘Big Beat Challenge’ grant of £30m for research into cardiovascular medicine was given to CureHeart.

An injectable cure for inherited heart muscle conditions that can kill young people could be available within a few years after an international team of researchers were announced as the winners of the British Heart Foundation (BHF).

The global award of £30m is one of the largest non-commercial grants ever given and presents a ‘once-in-a-generation opportunity to provide hope for families affected by inherited heart conditions.

The winning team, CureHeart, will seek to develop the first cures for inherited heart muscle diseases by pioneering revolutionary and ultra-precise gene therapy technologies that could edit or silence the faulty genes that cause these conditions.

The team – made up of world-leading scientists from the UK, US and Singapore – was selected by an International Advisory Panel chaired by Professor Sir Patrick Vallance, Chief Scientific Advisor to the UK Government.

The team will take the revolutionary gene-editing technology of CRISPR to the next level by deploying ultra-precise techniques, called, base and prime editing, in the heart for the first time. These ground-breaking approaches use molecules that act like tiny pencils to rewrite the single mutations that are buried within the DNA of heart cells in people with genetic cardiomyopathies.

Inherited heart muscle diseases can cause the heart to stop suddenly or cause progressive heart failure in young people. Every week in the UK, 12 people under the age of 35 die of an undiagnosed heart condition, very often caused by one of these inherited heart muscle diseases, also known as genetic cardiomyopathies. Around half of all heart, transplants are needed because of cardiomyopathy and current treatments do not prevent the condition from progressing.

It’s estimated that one in 250 people worldwide – around 260,000 people in the UK – are affected by genetic cardiomyopathies, with a 50:50 risk they will pass their faulty genes on to each of their children. In many cases, multiple members of the same family will develop heart failure, need a heart transplant, or are lost to sudden cardiac death at a young age.

BHF Professor Hugh Watkins, from the Radcliffe Department of Medicine at the University of Oxford and lead investigator of CureHeart, said: “This is our ‘once-in-a-generation’ opportunity to relieve families of the constant worry of sudden death, heart failure and the potential need for a heart transplant. After 30 years of research, we have discovered many of the genes and specific genetic faults responsible for different cardiomyopathies, and how they work. We believe that we will have a gene therapy ready to start testing in clinical trials in the next five years.

“The £30 million from the BHF’s Big Beat Challenge will give us the platform to turbo-charge our progress in finding a cure so the next generation of children diagnosed with genetic cardiomyopathies can live long, happy and productive lives,” he added.