A first in the world of science: Human DNA was edited directly inside the body

A historical threshold has been crossed in the field of medicine and genetics; This is the first time DNA inside the human body has been directly altered using a gene editing technique. This breakthrough, published in the peer-reviewed journal Nature, paves the way for faster, cheaper and less invasive genetic therapies to repair DNA in the human body by replacing complex laboratory procedures.

According to the Independent, in the clinical study, scientists used a high-precision "prime editing" tool to correct the genetic mutation in liver cells caused by Wilson's disease, a rare disorder that prevents the body from excreting excess copper. Wilson's disease is caused by mutations in the ATP7B gene, which produces the protein responsible for removing excess copper from the liver. If left untreated, accumulated copper can reach toxic levels and cause serious liver damage, nerve damage and neurological disorders.

While current treatments require the lifelong use of copper-scavenging drugs, gene editing technology has the potential to offer a one-time and permanent solution by directly addressing the problem caused by the change in the gene.

While standard gene editing methods such as CRISPR-Cas9 technologies generally knock out genes rather than rewriting them, primary editing works like a microscopic word processor, capable of highly precise additions, deletions and changes to specific sections of DNA. The treatment developed by the US biotechnology company Prime Medicine delivers gene editing tools to liver cells through tiny lipid nanoparticles, similar to the technology used in mRNA vaccines.

Editing DNA directly in the body eliminates the need for costly and risky processes such as removing a patient's cells, modifying them in the laboratory, and then reinjecting them. In traditional cell editing procedures, patients often need to receive heavy chemotherapy in order for the edited cells to be accepted into the body. Biotechnology analyst Joseph Thome of TD Cowen emphasizes that direct treatments could significantly reduce production costs, making them more widely available.

On the other hand, experts warn that in-body gene editing also poses its own challenges, as doctors cannot verify whether the edits are completely correct before the cells begin to proliferate. Delivering gene-editing tools precisely to target organs remains a fundamental challenge, says biomedical engineer Krishanu Saha of the University of Wisconsin-Madison.

Prime Medicine is expected to announce initial clinical results from this trial next year, along with decisions on other in-vitro CRISPR therapies currently awaiting regulatory approval.