First human gene therapy surgery attempts to halt common form of vision loss
By: Rich Haridy
In an extraordinary milestone procedure, scientists in the UK have performed the first gene therapy operation aimed at stopping progression of the most common cause of vision loss. The success of the procedure is yet to be determined, however the scientists suggest this one-off operation could be performed early in the degeneration process and essentially halt the disease in its tracks. 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 worldwide. The common degenerative disease begins with disruptions in a person’s central field of vision, and while it doesn’t always result in complete vision loss, it can profoundly alter one’s ability to undertake simple things such as reading or even recognizing faces.
In AMD, retinal cells are progressively destroyed by an aggressive immune response. This immune response has been found to be triggered by an overactive protein system. The experimental therapy is designed to deliver a gene that codes for a protein that essentially deactivates this aggressive immune response, and to do this the treatment utilizes a benign virus to transport the new gene to where it needs to go.
‘We’re harnessing the power of the virus, a naturally occurring organism, to deliver the DNA into the patient’s cells,” says Robert MacLaren, an ophthalmologist from the University of Oxford working on the project. “When the virus opens up inside the retinal cell it releases the DNA of the gene we have cloned, and the cell starts making a protein that we think can modify the disease, correcting the imbalance of the inflammation caused by the complement system.”
The therapy involves surgically detaching the retina and directly delivering the viral solution to the back of the eye. This targeted approach ensures the treatment is contained to a single point, while the virus is also engineered to only infect specific retinal cells….
Source: New Atlas