The British Heart Foundation announced the winner of its $36 million Big Beat Challenge, one of the largest non-commercial awards ever given for heart research.

The winning team, CureHeart, brings together researchers from the U.K., U.S. and Asia, including Eric Olson, professor and chair of the Department of Molecular Biology at UT Southwestern Medical Center.

Olson is the founding chair of the department and directs the Hamon Center for Regenerative Science and Medicine and the Wellstone Center for Muscular Dystrophy Research. He holds the Robert A. Welch Distinguished Chair in Science and the Annie and Willie Nelson Professorship in Stem Cell Research.

He has spent his career investigating heart and muscle development and disease, leading to his participation on the CureHeart team. The Olson Lab at UTSW has been incredibly successful in muscular research, most recently providing a new way to correct the mutation that causes Duchenne muscular dystrophy through gene editing.

CureHeart made the top of the list with its gene editing therapy aimed at curing inherited heart muscle diseases, known as cardiomyopathies.

A BHF release said the technology 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 deadly conditions.

The project will use gene-editing technology CRISPR to complete base and prime editing in the heart for the first time.

It works by correcting or silencing a faulty gene in the pumping machinery of the heart, either by re-writing the DNA at a single location or by switching off the entire copy of the faulty gene.

The technique was described as molecules that act like tiny pencils to rewrite the single mutations that are buried within the DNA of heart cells in people with heart conditions.

It can also help the heart produce enough proteins to function normally, again by fixing or stimulating the faulty gene.

With ultra-precise base editing technology, we hope to be able to correct a single letter and larger errors in the genetic code. This would mark a breakthrough for not only genetic cardiomyopathies, but for many heart conditions, said Olson in the release.

The project is the next step toward a real-world application, having already proved successful in animals with cardiomyopathies and in human cells. Members of the team believe therapies could be delivered through an arm injection, slowing or stopping the progression of cardiomyopathies, or even curing the disease entirely.

If successful, the research could have enormous impacts.

Every year in the US, around 2,000 people under the age of 25 die of a sudden cardiac arrest, often caused by one of these inherited muscle diseases, said the release. Current treatments do not prevent the condition from progressing, and around half of all heart transplants are needed because of cardiomyopathy.

The researchers believe it could also be successful in preventing the disease from being expressed if inherited. Children who receive the faulty gene from their parents could receive the injection and never develop cardiomyopathy in the first place.

Over the last 30 years, we have made extraordinary advancements in our understanding of the genetic mistakes that cause cardiomyopathy. CureHeart is a once-in-a-generation opportunity to transform this knowledge into a cure, said Olson in the release.

The technology is still in the research and development phase, but Olson said the funds will be used to optimize the method and expand it to a larger number of genetic diseases of the heart, and could move to clinical trials in the next few years.

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UTSW researcher part of team awarded $36 million heart research grant - The Dallas Morning News

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