It could be used to create a host of new drugs
in June marks what researchers are calling the first time CRISPR has been shown to treat a genetic disorder when directly administered to human patients.
“To imagine using [CRISPR] as a therapy for people, you need to figure out how to get these editing tools into the cells you’re trying to fix. That’s where messenger RNA comes in,” explains Daniel Anderson, a professor of chemical engineering at the Massachusetts Institute of Technology and a co-founder of CRISPR Therapeutics, which uses CRISPR technology to develop medications. Anderson was not involved in the research.
As Dr. John Leonard, Intellia’s president and CEO, puts it: “mRNA is a way to make CRISPR gene editing come alive. CRISPR is the workhorse; mRNA encodes it.” Chien has believed in mRNA’s drug-development potential for more than a decade. When Moderna was founded in 2010, in fact, its chief goal was to develop mRNA-based drugs, not vaccines. He continues to work on an mRNA-based drug he hopes could eventually treat heart conditions.
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