Genetic heart conditions could be cured for first time in ‘defining moment’

The world's first cure for genetic heart conditions is set to be developed by scientists who will rewrite the genes in order to fix them.

A global team of experts from the UK, US and Singapore are working together to design a jab in the arm to save thousands of lives.

For the first time, the team will use precision genetic techniques, called base and prime editing, in the heart for the purpose of designing and testing the first cure for inherited heart muscle diseases. The techniques have been used in animal studies.

Prof Sir Nilesh Samani is the medical director of the BHF.

sudden death or progressive heart failure can be caused by inherited heart conditions. There are 260,000 people in the UK who have the condition.

Twelve people under the age of 35 die of a heart condition every week in the UK, which is caused by an inherited heart disease.

People with genetic cardiomyopathies have a 50% chance of passing faulty genes on to each of their children, which can lead to heart failure, need a heart transplant, or death at a young age.

Prof Sir Patrick Vallance is the UK government's chief scientific adviser.

Prof Hugh Watkins from the University of Oxford is the lead investigator of the CureHeart project.

He said that this is a chance to relieve families of the constant worry of sudden death, heart failure and need for a heart transplant. Many of the genes and specific genetic faults responsible for different cardiomyopathies have been discovered. In the next five years, we believe we will have a gene therapy that is ready to be tested.

The aim of the research is to permanently correct or silence the genes that cause these heart problems.

The CureHeart project's co-lead is a professor of medicine at Harvard Medical School.

She said that most of themutations that they find in their human patients will change one letter of the DNA code. It's possible that we could change that single letter and restore the code to make a normal gene.

She said that the goal was to fix the hearts and possibly return them to normal function.

We may be able to deliver these therapies in advance of disease in individuals we know from genetic testing are at extreme risk of having disease development and progressing to heart failure. We have never before been able to deliver cures and that is what our project is about. We know we can do it, and we want to start.