Scientists win $36.5M for cardiac gene editing therapy

By The Science Advisory Board staff writers

August 1, 2022 -- An international team won 30 million pounds ($36.5 million U.S.) to develop cures for inherited heart muscle diseases that use ultra-precise gene therapy technologies to edit or silence the faulty genes that cause the conditions.

U.K., U.S., and Singapore scientists collaborated on CureHeart to snag one of the largest noncommercial grants ever given. They said they've discovered many of the genes and specific genetic faults responsible for different cardiomyopathies and how they work.

CureHeart uses CRISPR as well as base and prime editing to rewrite the single mutations embedded in the DNA of people with genetic cardiomyopathies. The new approach will correct or silence the faulty gene by rewriting it or switching off the entire copy of the faulty gene. Also, where the faulty gene does not produce enough protein for the heart muscle to work as it should, the team plans to increase the production of healthy heart muscle proteins.

The gene editing techniques could also keep cardiomyopathies from being passed down in families.

The team has already shown these approaches work in animal studies and with human cells. Now with the grant, they'll be able to ramp up research. They believe they'll have a gene therapy ready to start testing in clinical trials in the next five years.

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