A powerful gene-editing technique has been used to edit DNA directly in a person’s body — the first known instance of such a treatment.
The milestone treatment is part of a clinical trial of prime editing, a technique that can alter DNA with more precision than standard CRISPR–Cas9 methods can. In this case, prime editing was used to treat a condition called Wilson disease by rewriting DNA in liver cells, to erase a mutation that prevents them from eliminating excess copper.
Prime Medicine, a biotechnology company in Cambridge, Massachusetts, announced that it had treated the first person in its trial earlier this month. It expects to announce the first results from the study next year.
“I never dreamed that in my lifetime I would see this applied to my rare disease,” says Rhonda Rowland, president of the Wilson Disease Association in Atlanta, Georgia. “It’s going to be a really exciting year.”
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