The laboratory era in gene therapy is coming to an end: The era of direct DNA editing in the human body has begun

This technology, which has much higher sensitivity than traditional CRISPR methods, aims to save patients from long and difficult treatment processes by correcting the faulty gene in organ tissues on site.

HOPE OF A ONE-TIME TREATMENT AGAINST WILSON'S DISEASE

The historic treatment in question was tested on a patient with Wilson's disease, which causes dangerous copper accumulation in the liver. By targeting the mutation in the ATP7B gene, which prevents the body from excreting excess copper, scientists managed to delete the cellular-sized error directly inside the organ.

While the first patient of the clinical study conducted by the Massachusetts-based biotechnology company Prime Medicine is being treated, the first results from the research are expected to be made public next year. Rhonda Rowland, President of the Wilson Disease Association, said, "I never thought I would see this technology applied to my own disease in my lifetime," and stated that the development is a great source of hope for patients.

IN VIVO METHOD WHICH FINISHES THE LABORATORY PROCESS WILL REDUCE COSTS

In approved gene therapies applied to date, cells taken from the patient were first separated in a laboratory environment, DNA was edited and then reinjected into the patient. In the new approach, called "in vivo", there is no need to take the cells out.

Experts state that this method will create a major commercial and logistical revolution in the healthcare industry, and emphasize that treatments applied directly to the body will be much cheaper and faster, instead of personalized cell production. However, the method also has its own difficulties. Ensuring that gene regulatory molecules only reach the target organ and prevent them from damaging other tissues is one of the most critical issues that scientists work on.

WHILE THE RISK OF CHEMOTHERAPY IS ELIMINATED, THE CHALLENGE OF CONTROL IS DISCUSSED

While the accuracy of cellular changes in extracorporeal gene editing processes can be confirmed in the laboratory before transplantation to the patient, this control is not possible in in-body applications. On the other hand, experts point out that the vital risks caused by heavy chemotherapy processes applied to clean the patient's unedited cells in traditional gene transplants are eliminated with this method.

In vivo gene therapies, which are undergoing clinical trials in many different areas, from lowering blood cholesterol to lung diseases, are expected to enter standard treatment protocols in the coming years.