One genetic typing error in DNA destroys young lives
The disorder arises from a defect in the dystrophin gene on the X chromosome. At birth, the disease is not visible, but from infancy, motor problems develop. The children have difficulty standing up, and running and climbing stairs are also difficult. A wheelchair is indispensable from the age of 8. In addition, the disease also affects the heart and breathing, which is ultimately fatal.
Gene therapy research gives hope
The road to a cure is still long, but VUB research on gene therapy is showing hopeful results. Gene therapy allows disease-bearing genes to be replaced by healthy versions of the gene. For more than 30 years throughout their scientific careers, Prof. Dr. Thierry VandenDriessche and Prof. Dr. Marinee Chuah, of the Department of Gene Therapy and Regenerative Medicine, have been committed to developing a treatment for hereditary diseases through gene therapy. The research team sought a means to introduce into the genetic material of muscle cells a copy of a functioning dystrophin gene, which takes over the function of the defective gene.
To activate the healthy gene, they developed a body's own molecular turbo engine that significantly improves the efficacy and safety of gene therapy. This prevents muscle deterioration and is an important first step toward further clinical applications. Moreover, the technique can also be used for other inherited muscle disorders such as Steinert's disease.
Cycling Against Duchenne
Initiator Luc Vandevyvere is the proud grandfather of his grandson Stan. When he learned the news that baby Stan was diagnosed with Duchenne, his joy first gave way to fear and then to hope: "As a grandparent, I still have a hard time coming to terms with this terrible news. I can safely say that I have two lives: my life before and my life after Duchenne.".
Hoping to find some peace and make peace with his grandson's inevitable fate, Luc launched Cycling Against Duchenne in 2020. That year, he cycled 2,000 km around the Benelux, a year later he added another 2,700 km and in 2022 he cycled all the way to Rome (2,850 km!). With his bike rides he wants to raise money for the benefit of the VUB research on gene therapy. The project already raised more than 22,000 euros!
Duchenne Parent Project
Duchenne Parent Project Belgium is an independent association of parents and family members of children with Duchenne muscular dystrophy. Because their children could use all the help they can get, they want to work together to get scientists, doctors, companies and the government moving and to accelerate research into a treatment or cure for Duchenne. Duchenne Parent Projects exists worldwide and is a member of the World Duchenne Organization (WDO). The association organizes a variety of activities to benefit research.
More research is needed to save young lives
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