Prime Medicine edits DNA inside the human body for the first time in Wilson disease trial

US biotechnology company Prime Medicine used a technique called prime editing in a clinical study to correct a genetic mutation that causes Wilson disease in liver cells. The work is described as the first direct change of DNA inside the human body using gene editing. Wilson disease is a rare condition that prevents the body from removing excess copper; if untreated, copper can build up to toxic levels, causing serious liver damage and nerve and neurological problems. The findings were published in the peer-reviewed journal Nature.

The treatment delivers gene editing tools to liver cells through tiny lipid nanoparticles, a technology similar to that used in mRNA vaccines. It removes the need to extract a patient's cells, edit them in a laboratory and inject them back, processes that are costly and risky. Traditional cell editing often requires patients to undergo heavy chemotherapy. Current treatments need lifelong copper-lowering medication, while gene editing could offer a one-time, lasting solution.

Experts caution that doctors cannot verify whether edits are fully accurate before cells begin to multiply, and that delivering editing tools precisely to target organs remains a 'fundamental challenge', according to Krishanu Saha of the University of Wisconsin-Madison. Prime Medicine is expected to announce its first clinical results from the trial next year.