The British Heart Foundation has announced an award of £30 million ($36 million) over 5 years to an international team to develop gene-editing treatments for inherited heart diseases.
When a high school athlete dies from a heart attack or a young woman needs a heart transplant, it is often because they inherited a DNA mutation that causes heart muscle disease. Today, in the biggest research grant ever from the research charity, the British Heart Foundation announced an award of £30 million over 5 years to an international team to develop gene-editing treatments for these deadly diseases.
Researchers know many of the mutations behind these diseases, suggesting one-time injections of gene treatments could cure them. But CureHeart scientists face major challenges, Watkins says. Some defective muscle heart genes are very large, making it impossible to use a virus to ferry in a good copy. In addition, heart researchers may need to correct only the bad copy of a gene and leave a healthy copy untouched, which rules out the classic CRISPR tool that edits by cutting both DNA strands.
, a disease that causes children to age rapidly. Harvard chemist David Liu, whose lab developed base editing, is part of CureHeart. He’s joined by muscle gene-editing and heart disease experts in the United States, United Kingdom, and Singapore.
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