In a world first, scientists rewrite DNA to cure 'genetic heart conditions'

"Our once-in-generation opportunity..."
Mert Erdemir
An illustration of DNAmesut zengin/iStock

An international team of scientists from the U.K., U.S., and Singapore is working together to develop an injectable cure for genetic heart conditions by rewriting DNA. The team named CureHeart has been awarded a £30 million grant from the British Heart Foundation (BHF).

The researchers will employ precision genetic techniques in the heart for the first time with the aim of silencing defective genes and develop and test the first treatment for genetic heart diseases. Animal tests had already proven before that the techniques work.

"This is a defining moment for cardiovascular medicine," said Professor Sir Nilesh Samani, the BHF’s medical director. "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-in-a-generation opportunity

Genetic cardiomyopathy diseases affect about 260,000 people only in the U.K., and they can cause sudden death or progressive heart failure at any age. Considering the fact that the risk of these faulty genes to pass on to each of their children is 50/50, it can be said that multiple members of the same family will develop heart failure, need a heart transplant, or die at a young age.

"This is our once-in-generation opportunity to relieve families of the constant worry of sudden death, heart failure, and potential need for a heart transplant," said Professor Hugh Watkins from the Radcliffe Department of Medicine at the University of Oxford and lead investigator of CureHeart. "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".

Silencing the faulty genes

The aim of the new research is to permanently correct or silence the faulty genes responsible for these cardiac issues.

"Acting on our mission will be a truly global effort," said Dr. Christine Seidman, a professor of medicine at Harvard Medical School in the U.S. and co-lead of the CureHeart project. "We’ve brought in pioneers in new, ultra-precise gene editing, and experts with the techniques to ensure we get our genetic tools straight into the heart safely. It’s because of our world-leading team from three different continents that our initial dream should become reality."

By creating the world’s first genetic cure for heart disease, the study could make a giant leap in cardiovascular medicine. It definitely is a promising step towards stopping families from losing loved ones to these cruel diseases. However, the team also states that they need continuous support to achieve such a milestone for the millions of people around the world living with genetic heart disease.

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