UTSW researcher on team awarded $36 million cardiac research grant
The British Heart Foundation has announced the winner of its $36 million Big Beat Challengeone of the largest non-commercial prizes ever awarded for cardiac research.
Winner team, CureHeartbrings together researchers from the UK, US and Asia, including Eric Olson, Professor and Head 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 Chair of Scientific Distinction and the Annie and Willie Nelson Chair in Stem Cell Research.
He has spent his career studying heart and muscle development and disease, which led him to participate in the CureHeart team. The Olson Laboratory at UTSW has had incredible success in muscle research, most recently providing a new way to correct the mutation that causes Duchenne muscular dystrophy through gene editing.
CureHeart topped the list with its gene-editing therapy aimed at curing inherited diseases of the heart muscle, known as cardiomyopathies.
A statement from BHF said the technology “will seek to develop the first cures for inherited heart muscle diseases by pioneering breakthrough, ultra-precise gene therapy technologies that could edit or silence the faulty genes that cause these deadly conditions.” .
The project will use CRISPR gene-editing technology to complete core and core editing in the heart for the first time.
It works by fixing or silencing a faulty gene in the pumping machinery of the heart, either by rewriting DNA in a single location or by switching off the entire copy of the faulty gene.
The technique has been described as “molecules that act like tiny pencils to rewrite unique mutations that are buried in the DNA of heart cells” in people with heart disease.
It can also help the heart produce enough protein to function normally, again by fixing or stimulating the faulty gene.
“With ultra-precise base-editing technology, we hope to be able to correct single letter and larger errors in the genetic code. This would mark a breakthrough not only for genetic cardiomyopathies, but also for many heart diseases,” Olson said in the statement.
The project is the next step towards real-world application, having already been proven in animals with cardiomyopathies and in human cells. Team members believe the therapies could be delivered through an injection in the arm, slowing or stopping the progression of cardiomyopathies, or even completely curing the disease.
If successful, the research could have huge impacts.
“Each year in the United States, approximately 2,000 people under the age of 25 die from sudden cardiac arrest, often caused by one of these inherited muscle diseases,” the statement said. “Current treatments do not prevent the disease from progressing, and about half of all heart transplants are needed because of cardiomyopathy.”
Researchers believe it may also be successful in preventing the disease from expressing itself if it is hereditary. Children who receive the defective gene from their parents might receive the injection and never develop cardiomyopathy in the first place.
“Over the past 30 years, we have made extraordinary progress in our understanding of the genetic errors that cause cardiomyopathy. CureHeart is a once-in-a-generation opportunity to turn this knowledge into a cure,” Olson said 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 more genetic heart conditions, and could move into clinical trials in the next few years. .