Scientists have used a high-precision gene-editing technique to alter DNA directly inside a human body for the first time, aiming to cure a rare liver disorder.
According to a report by The Independent, researchers deployed a tool called "prime editing" in a new clinical trial to correct a genetic mutation responsible for Wilson disease.
Wilson disease prevents the body from clearing excess copper, leading to toxic build-ups that cause severe liver damage, nerve impairment, and neurological issues.
The trial's findings, published in the journal Nature, point towards a future of cheaper, faster, and less invasive genetic therapies.
By operating directly inside the body, the method bypasses the costly and risky process of extracting, modifying, and re-injecting patient cells, The Independent reports. It also spares patients from the harsh chemotherapy traditionally needed to make room for newly edited cells to take hold.
While standard gene-editing procedures like CRISPR-Cas9 typically disable genes, prime editing functions as a microscopic word processor that can precisely insert, delete, and replace specific DNA segments.
US biotechnology firm Prime Medicine developed the treatment, using tiny lipid nanoparticles to transport the editing tools into liver cells.
Currently, Wilson disease requires lifelong copper-clearing medications. The new therapy offers the prospect of a single, permanent repair to the faulty ATP7B gene.
"I never dreamed that in my lifetime I would see this applied to my rare disease," Rhonda Rowland, president of the Wilson Disease Association, was quoted by the British newspaper as saying.
However, experts have warned that in-body editing carries distinct risks, as doctors cannot verify the accuracy of the edits before the cells replicate.
Krishanu Saha, a biomedical engineer at the University of Wisconsin-Madison, told the publication that safely delivering the tools to target organs remains "the core challenge."
Initial clinical results from the trial are expected next year.