Stop blindness in children!

Imagine that you or your child's vision becomes increasingly poor at birth or in the years that follow. Reading, recognizing faces, finding your way independently is impossible and ultimately there is a good chance that you will become blind. This is what happens in the case of many hereditary eye diseases. In the Netherlands, more than 5.000 people are affected by these eye diseases, often at a young age. These eye diseases affect as many as 2.5 million people worldwide. At Amsterdam UMC, top researchers want to develop innovative gene therapy treatments to prevent blindness caused by these hereditary eye diseases. No treatment is possible at this time. You can contribute to this research.

This type of research is innovative, experimental, complex and very expensive. These studies can partly be set up with subsidies from regular subsidy providers. But this is not nearly enough to further develop these promising treatments and ultimately apply them to patients.

Error in the DNA

A hereditary eye disease involves an error in the DNA (a gene) that is essential for the normal functioning of the eye. The researchers think that this defective gene can be repaired with 'gene therapy'. The genetic error is corrected through a repair. If the eye cells are already too damaged, it could be possible in the future to replace them with new cells: so-called stem cells. Despite the many experimental developments in this area, there is still no treatment for almost all serious hereditary eye diseases. We want to change that.

Why Amsterdam UMC?

Amsterdam UMC houses a world-famous gene and stem cell therapy laboratory that works with the latest insights into gene therapy and stem cells. This laboratory has the ideal infrastructure and knowledge to develop new forms of gene therapy and stem cell treatment for serious hereditary eye diseases. To simulate the diseases as closely as possible, disease models are made from cultured cells of the patients themselves, on which new gene and stem cell treatments are tested. The most promising treatments are then further developed for actual use in patients.

Prof. Dr. Camiel Boon

The research is conducted under the supervision of Prof. Camiel Boon. Boon is an ophthalmologist and, at the age of 36, was the youngest professor of ophthalmology in the past 40 years and was appointed professor of Ophthalmology. He focuses primarily on the diagnosis and treatment of retinal diseases and conducts research into treatments for these serious eye diseases. He works closely with other experts in the field of the development of these treatments in Amsterdam UMC and beyond.

'Every week I am confronted with the impact of a hereditary eye disease on children and young adults. These eye diseases can lead to blindness within a few years. With this research we want to do everything we can to prevent blindness in children in the future.'Prof. Dr. Camiel Boon

Support the research

Your support makes it possible to develop gene and stem cell therapy for hereditary eye diseases. This research is important for children and young adults affected by blinding hereditary eye diseases. But elderly people with eye diseases also benefit from these new insights. These insights could even have an impact on better and new treatments for other diseases.