Six Ways CureHeart Is Set to Change the World

For most of the 21st century, the silicon computer chip has governed the way we live our lives. These tiny electronic cerebrums power the smartphones that we’ve become surgically attached to. They now drive our trains, fly our planes and—thanks to the microchips under their skin—they even allow our pets to board VistaJet flights too. But it could be argued that humanity is on the cusp of another new epoch: The Age of the molecule.

The last decade has seen a spate of discoveries regarding DNA, the central molecule through which genetic information is stored. This is no mean feat, as the width of a DNA double helix is about 50,000 times smaller than the thickness of a human hair.

In 2012, two molecular biologists, Jennifer Doudna and Emmanuelle Charpentier, developed a technique of gene-editing known as CRISPR. It works something like this: a ‘guide molecule’ called a ‘guide RNA’ associates with the Cas9 enzyme to be able to find the sequence on DNA to cut or edit.

When the cell attempts to mend this dam- age, it usually ends up destroying the gene. Scientists can also cut, edit and paste parts of the mutated DNA just like a “pair of molecular scissors,” as Scientific American has put it. They can also replace those faulty genes responsible for genetic disorders with healthy strands of DNA, injecting them alongside CRISPR molecules. Doudna and Charpentier have since won the Nobel Prize in Chemistry for their work. While scientists tampering with our genetic code raises all manner of social and ethical considerations (designer babies for one), it’s been predicted that CRISPR and the new DNAediting possibilities that have followed may one day be able to cure diseases caused by genetic mutations, including congenital blindness, sickle-cell anemia, cystic fibrosis and perhaps even some cancers. In other words, genetic disease could be wiped out for good.

A cure for debillitating and often lifethreatening inherited heart muscle diseases— which cause suffering for millions of families across the world—is also something firmly within science’s grasp, thanks to gene editing. Here, we look at the ways one project, CureHeart, could change the world.

It’s estimated that one in 250 people worldwide are affected by an inherited heart muscle disease. These conditions, also known as genetic cardiomyopathies, cause the heart to become progressively more damaged over time. Many with the condition develop heart failure or need heart transplants, often at young ages. Sudden cardiac death is a possible outcome, again with otherwise healthy young people often affected.

Yet, an end to the suffering caused by some genetic cardiomyopathies could well be in sight within a decade.

CureHeart is a global project led by scientists from the UK, US and Singapore and funded by a £30m/$37m grant from the British Heart Foundation (BHF). It’s currently designing and testing the first ever cures for inherited heart muscle diseases, which could be administered to patients via an injection.

A jab to the arm seems like a simple solution, sure, but the science behind gene-editing is incredibly complex. If CRISPR are “molecular scissors,” CureHeart will apply a newer technique called “base editing,” which has been likened to a molecular pencil that can correct single-letter errors in faulty genes within the heart cells of people with genetic cardiomyopathies.

CureHeart aims to have one or more treatments ready for testing in early-stage clinical trials within the next five years.

“[CureHeart will] hopefully achieve something I never thought would happen in my working career: to come up with the first cures for these conditions,” says Hugh Watkins, lead investigator on the CureHeart project and BHF Professor of Cardiovascular Medicine at the University of Oxford. “It’ll have a transformational impact on the families we’ve looked after for such a long time.”