Two different papers published in the newest issue of Nature focus on experimental gene therapies to potentially repair the genes that cause hypertrophic cardiomyopathy. This research even caught the eye of Dr. Eric Topol, a prominent cardiologist at the Scripps Institute whose focus on genetics and digital technologies aims to change the future of medicine.
Researchers in the first study were able to correct pathogenic HCM mutations in an MYH7 gene. This was done both in cardiomyocytes (the cardiac cells which cause the heart to contract) derived from human pluripotent stem cells, as well as in mice with human-like HCM mutations.
The second study used two different genetic therapies to prevent HCM caused by the R403Q mutation in more than 70% of cardiomyocytes carrying the mutation.
The researchers hope that these findings will soon allow scientists to treat patients who carry an HCM gene BEFORE the disease actually develops, thereby interrupting the cycle and preventing the disease
It would be very exciting to see a clinical trial testing this technology. These therapies show great promise for our children and grandchildren.