Gene therapy risks serious complications as modified viruses trigger unexpected issues in young patients, raising safety concerns.
Gene therapy faces safety concerns
Gene therapy, which has already helped children with rare genetic diseases, is now under scrutiny due to serious complications in some young patients, according to an October 16, 2026, report by The Atlantic.Complications linked to virus use
The investigation suggests that the complications may involve the virus used to deliver corrective genes. These issues, though rare, have affected children with severe genetic conditions. The therapy often uses adeno-associated viruses (AAV), which can be harder to control than expected.Cases in China and the US
In China, a child in a clinical trial died from acute respiratory distress syndrome, where the lungs filled with fluid and lacked oxygen. Another child died from blood clots that blocked blood flow to vital organs. In Philadelphia, a five-year-old boy with Hurler's disease developed a brain tumor after the virus used in his treatment inserted itself into his DNA.Viral risks in gene therapy
The AAV virus used in these treatments is modified in the lab to prevent replication or infection, acting as a carrier for the functional gene. However, the viral component can behave unpredictably, sometimes integrating into the patient’s DNA, which led to the brain tumor in the American boy.
