Born With a Broken Gene? One Shot Is Being Tested to Fix It for Good.

Prime Medicine has given one infusion of a DNA rewriter to a Wilson disease patient; about 42 people will test whether a single shot can replace a lifelong drug, with first data due in 2027.

TL;DR

A new kind of gene editor is now working inside a human body for the first time. Prime Medicine has given one infusion of a "prime editor" to a person with Wilson disease, an inherited condition in which the body cannot clear copper, and Nature news calls it the first known use of prime editing inside a person. A prime editor rewrites a misspelled DNA letter in place, without cutting both strands of the DNA, which is why researchers see it as a gentler fix. No results exist yet; the first data are due in 2027. But the question the trial asks is the one that matters: can a single shot replace a pill people would otherwise take for the rest of their lives?

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