Prime editing rewrites DNA inside human body for the first time

Scientists in a breakthrough study have used prime editing to rewrite DNA directly inside a patient’s body for the first time, uncovering new possibilities for treating genetic diseases by correcting harmful mutations.

Prime Medicine dosed the first person in a trial of PM577-a prime editing therapy that corrects a faulty gene directly inside the body. The study was published in the journal Nature facilitates the development of high-efficiency, cost effective and minimally invasive genetic interventions that repair DNA inside the human body, replacing complex laboratory procedures.

Wilson disease is caused by mutations in the ATP7B gene which produces a protein responsible for removing surplus copper from the liver.

Developed by US technology firm Prime Medicine, the treatment shuttles gene-editing tools into liver cells using lipid nanoparticles.

Conventional cell-editing procedures often subject patients to rigorous chemotherapy to clear room in the body for edited cells to take hold.

Experts caution that editing inside the body comes with its own difficulties as doctors cannot verify that edits are entirely accurate before the cells begin replicating. Prime Medicine is slated to publish initial clinical data from the study next year, alongside decisions on other in-body CRISPR treatments currently pending regulatory approval.