This is the last resort for Roel Keynes (33). Either this treatment will work and he will recover, or he will go blind within the next ten years. In retinitis pigmentosa, the light-sensitive cells do not function properly due to an error in the gene, which eventually causes these cells to break down. During treatment, a properly working gene is injected under the retina, so that a good gene can replace a bad gene. “The results are so good so far, my sight is completely back,” he tells Editie NL. “I had it in mind that I would go blind, but thanks to this treatment I have a whole future ahead of me again.”
And ophthalmologist Susan Yeezer of Radbodomec is very proud of that. Treatment development has been a long-term process. “Scientists from all over the world are working on this. Eight patients were operated on all over the world before we started in the Netherlands,” she told Edie NL. Patients 9 and 10 are now undergoing surgery in the Netherlands, Rowell being one of them.
“What we do is we add a piece of a gene that works well to a virus from which we have removed the disease-causing part,” Yzer continues. “We inject this under the retina and then the virus penetrates into the cells. Then the properly working gene takes over the functions of the broken gene.”
stay independent
There is currently no other treatment for patients with this disease. Therefore, Rowell is very happy that he can participate in this study. “I always thought I was going to lose my sight, but now I just know I can stay independent for the rest of my life and that’s really cool.”
In the Netherlands, 1 in 3,000 people suffer from retinitis pigmentosa in the eye. This disease is caused by a fault in a gene.
Source: Al Ain Fund
Gene therapy is being applied to many genetic diseases and it could mean a lot for us in the future. Nils Reynders is conducting research into gene therapy for Alzheimer’s disease at Utrecht University. For this he uses an artificial virus. “This virus carries the gene that can cause cells in the brains of Alzheimer’s patients to produce a protein that protects the cell from Alzheimer’s disease.”
experimental
This seems hopeful, although according to Reinders, it will take years before it is implemented. “I think we’ll be working on this for another five to twenty years. The problem with gene therapy is that it’s very new, so there’s still a lot of testing to do in terms of safety. It’s still very experimental, but it’s promising that in the coming decades it will become Incurable diseases are curable.”
Whether gene therapy for the eye disease retinitis pigmentosa will actually work will be investigated in the coming years.
