Hypertrophic Cardiomyopathy: $36M Grant Targets Genetic Fix Research

Just Announced:  The University of Texas Southwestern Medical School (UTSW) is privileged to be a part of the team just awarded one of the largest non-commercial grants ever given for heart research, in this case for gene editing therapy for hypertrophic cardiomyopathy (HCM).

The British Heart Foundation’s Big Beat Challenge has granted the winning team Cure Heart, composed of researchers from the UK, US and Asia.  UTSW is most prominent in the field of molecular biology and the Department was founded and is chaired by Eric Olson, MD, whose work with colleagues there has been incredibly successful, most recently providing a new way to gene-edit and correct the mutation the causes Duchenne muscular dystrophy.

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.

Dr. Olson said in a release, “This would mark a breakthrough not only for genetic cardiomyopathies, but for many heart conditions.  Every year in the US, around 2,000 people under age 25 die of a sudden cardiac arrest, often caused by one of these inherited muscle diseases.  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 genetically expressed if inherited.  Children who receive the faulty gene from their parents could receive the injection and never develop HCM in the first place.

The technology is still in the R&D 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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