Gene Therapy: Potentially Restoring a Child’s Vision

There is a bad disease that manifests itself in childhood, it is called retinal dystrophy, can lead to blindness. However, for almost a year gene therapy, which aims to correct possible DNA errors, the culprits of the disease. In 2021, 10 children managed to restore their sight with this type of surgery. Here comes some good news from the Policlinico di Bari: an 11-year-old girl underwent risky surgery to regain her sight.

In order to simplify the very complicated process, in practice the child was given a healthy copy of the defective gene, in order for the whole organ to function properly. To get to the location of the device, gene was inserted under the retina with a adenovirus. The latter is nothing more than a pathogenic acting uselessly as a Trojan horse. Then, once inside, he frees the real therapy, which is a drug that contains the right piece of DNA.

Gene therapy is the future of medicine. Achieving this result required the combined effort of the operating room medical genetics, pharmacy, anaesthesiologists, clinical risk, health management and rare disease center of the outpatient clinic, as well as the entire team of the operating room ophthalmology. . After all these efforts, we have opened a new opportunity for patients suffering from retinal dystrophy lacking the hRpe65 gene.

Professor Giovanni Alessio, coordinator of the Policlinico di Bari . team

This hospital is one of six Italian centers authorized for this type of operation. The young patient will be monitored continuously to make sure vision has been restored correctly. Here’s another testimonial to how gene therapy can beat retinal dystrophy. In a day, this disease may just be a bad memory.