Science and education
Hurler’s disease is a serious, rare metabolic disease
The research project Eye for the Future has received support from the For Wis(h)dom Foundation. Researchers will use this money to prevent blindness in children with a rare metabolic disease.
For Wis(h)dom Fonden is a foundation that wants to make a meaningful contribution to research into the treatment of rare diseases. Thanks to this donation, WKZ will be able to research a treatment for blindness in children with Hurler’s disease in the coming years. The research project starts this year.
Hurler’s disease is a serious, rare metabolic disorder. In the Netherlands, there are currently around 25 children with this disease. Hurler’s disease affects your brain, your skeleton and your eyes. The brain damage can be stopped by a stem cell transplant. Researchers want to know if they can also treat damage to other body parts with a stem cell transplant. Unfortunately, it seems that the eye disease has not stopped in teenagers who have had a successful stem cell transplant. In these teenagers, the retina is still deteriorating. As a result, they are often increasingly night blind and visually impaired. There is no treatment for this eye disease.
In this project, the researchers are looking for a treatment for the eye disease. They do this by mimicking the disease in zebrafish and then treating the fish with different types of gene therapy. The results of the treatments are compared with each other to find the most effective treatment. At the same time, the researchers are mapping how the eye disease progresses. In this way, we can better understand how the disease develops and what consequences this has for a person’s vision. That information is also important for future studies of treatments for Hurler’s disease. This project is the first step in finding an effective treatment for the eye disease.
The research is carried out under the direction of Dr. Peter van Hasselt, pediatrician metabolic diseases at Wilhelmina Children’s Hospital and head of the follow-up program after stem cell transplantation for metabolic diseases (including Hurler’s disease). The research team also consists of Dr. Gijs van Haaften, associate professor and expert in zebrafish research in ultra-rare diseases, and Prof. Dr. Mies van Genderen from UMC Utrecht and Bartimeus. She is an ophthalmologist and specializes in rare retinal diseases. Hurler experts from collaborative centers in Manchester and Paris will be involved in the research.
Friends UMC Utrecht & Wilhelmina Children’s Hospital
These studies can start thanks to the support of the For Wis(h)dom Foundation to Friends UMC Utrecht & Wilhelmina Children’s Hospital, the charity of the (children’s) hospital. Would you like to know more about how you can promote research into new treatments at UMC Utrecht? Look here.
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Money for research against blindness in children
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