New therapies against blindness | Watch for news

New therapies against blindness | Watch for news
New therapies against blindness | Watch for news

Monday, April 1, 2024 – 11:21

Blindness can arise in different ways. In young people, a hereditary retinal disease is the main cause, for the elderly it is age-related macular degeneration. The Ministry of Education, Culture and Science is now investing 22 million euros in the Lifelong Vision project, in which researchers will develop new therapies that can be used for various forms of blindness.


An important part of the project is gene therapy. If an error in a gene leads to blindness, doctors can already introduce a completely new gene. They inject it under the retina into the eye, in the hope that it reaches the right cells and ensures that those cells start making healthy proteins again.
“Unfortunately, this does not work very well yet,” says Professor of Epidemiology and Genetics of Eye Diseases Caroline Klaver of Radboud University Medical Center, and leader of the consortium: “Many genes are not suitable for this because they are too large. We therefore want to carry out the repair much more precisely, with gene editing. We only rewrite the error in the gene, and therefore no longer replace the entire gene.”
Camiel Boon, professor of ophthalmology and clinical ophthalmogenetics at Amsterdam UMC, adds: “In the lab of Amsterdam UMC we will develop gene editing, also known as CRISPR/Cas. In the research group of Arthur Bergen, professor of Genetic Eye Diseases, we develop new disease models to optimally test these new treatments. And with the group of professor of AI and health Clarisa Sánchez, artificial intelligence is used to investigate who is most suitable for these innovative treatments. This way, the right patient receives the right treatment at the right time.”


The researchers are also looking at how they can protect cells in the eye. It has already been extensively mapped out why a cell in an eye with a genetic defect dies. This offers starting points for keeping cells alive, for example with a special cocktail of proteins that help the cells with this. And the strategy of zebrafish is also very interesting: they can repair their retina themselves, something that humans cannot do. The researchers want to see how zebrafish do this and find out whether this can also be introduced to humans.
The researchers will also develop cell therapy. “The retina is brain tissue. It is a complex tissue of up to ten different layers, in which the nerve cells all interact with each other,” says Klaver. “We previously tried to repair damaged retina by injecting stem cells, but that did not work well. We are now going to see if we can make a retina with a bio-printer that places the different cell layers on top of each other. And we are finding out how that printed retina can connect to the choroid. This is very new and great research.”

The article is in Dutch

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