First gene therapy operation in the world to end the common form of vision loss

Age-related macular degeneration (AMD) is the most common cause of vision loss in people over the age of 50 and affects millions of people around the world. The current degenerative disease begins with a disruption of a person's central field of vision. Although it does not always result in complete vision loss, it can profoundly alter the person's ability to do simple things like reading or even recognizing a face.

In AMD, retinal cells are progressively destroyed by an aggressive immune response. This immune response was found to be triggered by a hyperactive protein system.

Gyroscope Therapeutics, a UK-based company in collaboration with Oxford University, performed the first gene therapy operation aimed at stopping the progression of the most common cause of vision loss. The procedure can potentially deliver a gene that encodes a protein that essentially disables this aggressive immune response.

However, scientists still have to determine the success of the procedure. But, they suggest that this unique operation could be performed early in the process of degeneration and stop the disease in its wake.

Robert MacLaren, an ophthalmologist from Oxford University working on the project, said, "We are exploiting the power of the virus, a natural organism, to deliver DNA into the patient's cells. When the virus opens up inside the retinal cell, it releases the DNA from the gene we cloned and the cell starts to make a protein that we think can alter the disease, thus correcting the imbalance of inflammation caused by the complement system. "

For this method, scientists have used a benign virus to carry the new gene where it needs to go. It also involves surgically detaching the retina and administering the viral solution directly to the back of the eye. This targeted approach ensures that the treatment is confined to a single point, while the virus is also designed to infect only specific retinal cells.

The first person to undergo the procedure was Janet Osborne from Oxford. Like many people with AMD, it has the condition in both eyes, but is more advanced to the left eye. As it is typical of this disease, the central vision in his left eye has deteriorated and is very cloudy, although his peripheral vision is better.

At 80, her restricted vision makes housework such as preparing vegetables and sewing difficult, and she can not read very long. She often has trouble recognizing faces.

She says her motivation for participating in the trial was the opportunity to help others with AMD: "I did not think about myself. I thought of other people. For me, I hope my site is not going to be worse. It would be fantastic. This means that I would not be such a nuisance for my family. "

Although other clinical trials are needed to determine the degree of safety of the treatment, it will take some time before it is clearly proven to be effective. However, the scientists behind this incredible innovation hope that this unique gene therapy procedure can be administered at the early stages of AMD, stopping the disease before it begins to permanently damage the disease. eye and potentially saving the sight of millions of people around the world. world.

Professor MacLaren said, "This field is changing rapidly. Since we know a lot more about how treatment is produced and the effects of the virus on gene therapy in the back of the eye, as well as all the other gene therapy programs under development, I hope that we will see a treatment for people with dry AMD in the next few years. "