SAN DIEGO (CNS) - The Scripps Translational Science Institute will take part in groundbreaking genomic research into heart disease, funded by a $7.9 million grant by the National Institutes of Health that was announced Friday.
Scientists will use pluripotent stem cells -- non-embryonic stem cells -- to help them understand how a part of the genome called a "gene desert" is linked to the risk of developing heart disease. They hope to learn what takes place in the region, where no genes are present, to cause cells to become diseased.
"We're trying to figure out for the first time how this region works and which other parts of the genome or genes it's interacting with to make some people's cells become diseased," said Dr. Eric Topol, the study's principal investigator and director of STSI -- which is part of Scripps Health.
The researchers plan to use the pluripotent stem cells to recreate the artery lining of the heart for 2,000 participants, half of whom have developed coronary artery disease and the rest having lived to 80 without heart problems.
Once they figure out which genes are interacting with that gene desert region, they can use gene editing to remove damaged cells and, potentially, develop drugs to cure the disease, according to STSI scientists.
Topol, who carries a genetic risk of heart disease, plans to be the first person to have their heart artery lining cells genomically edited, which he described as being like "science fiction."
The experiments could help overcome a deficiency in genomic studies, in which researchers can find a problem area but don't understand why things are happening or uncover the specific offending gene, he said.
"Genome editing will allow us to edit each one and analyze which ones are the culprits," Topol said.
Sangamo BioSciences of Richmond, Calif., will share in the grant and assist with the research.
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