Briefs · Emerging Technologies
CRISPR Prime Editing Rewrites DNA in the Human Body for the First Time
Gene editing has gone inside the body. Prime Medicine has dosed the first patient in a trial of prime editing for Wilson disease, a condition where copper builds up and damages the liver, nerves and brain. The editor, PM577a, travels in liver-targeted lipid nanoparticles and rewrites DNA in liver cells. Correcting just 20–30% of them may be enough to help. In monkeys, one infusion edited half of liver cells. In mice, it corrected 80% with minimal off-target edits. The verdict is precisely prime: a word processor that could fix up to 89% of disease-causing variants, one infusion at a time.
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