A defanged form of CRISPR, which doesn’t slice or nick DNA, but rather alters the epigenome — the layers of chemical coding that sit on top of DNA and control the activity of genes — has aced its first substantive test.
When researchers used CRISPR “epigenome editing” to dial down a cholesterol-associated gene in monkeys, the animals’ blood levels of heart-disease-causing LDL, or “bad” cholesterol, plummeted by more than 50%, Jennifer Kwon, senior scientist at Tune Therapeutics, announced on Friday at the American Society of Gene and Cell Therapy meeting in Los Angeles.
The results, from Tune’s experiments in five cynomolgus monkeys, are the first published data showing successful CRISPR epigenome editing in a non-human primate; in the past few years, there have been similar successes in mice. It’s exciting news for Tune, which was founded in 2021 and is located in Durham, N.C., and Seattle, but also for the other young startups in this field trying to develop a new class of precision medicines that can write or erase epigenetic marks involved in human disease.
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