Learning the root defect in the genome would open the door to potentially developing new drugs, or identifying existing ones that could help a person's cells revert away from the path of developing a heart attack or coronary artery disease later in life.
Scripps Health will supply the samples and provide the genomic characterization of the cells. The Scripps Research Institute will create the induced pluripotent stem cells, under the leadership of chemical biologist Sheng Ding (who recently joined the Gladstone Institutes in San Francisco). ZFN-mediated genome editing will be performed by Sangamo BioSciences under the supervision of lead investigator Fyodor Urnov.
According to Dr. Topol, this study will address the biggest deficiency in genomics today. "We don't know the so-called functional genomics," he said. "We only know there's this zip code in the genome that's a problem spot, but we don't know what's going on in this zip code of hundreds of thousands of letters. We don't know which is the offending letter or group of letters. Genome editing will allow us to edit each one and analyze which ones are the culprits."
This "disease in a dish" approach to research has been employed with rare diseases in recent years, but the Scripps initiative marks the first time the combination of disease in a dish and genome editing are being applied for a common health condition.
"You can't possibly test the full impact of someone's genetics by doing these experiments in mice," said Levy. "A mouse's geno
|Contact: Mika Ono|
Scripps Research Institute