Máxima-researchers dr. Ruben van Boxtel en prof. dr. Hans Clevers – together with prof. dr. Edward Nieuwenhuis, prof. dr. Annelien Bredenoord and dr. Sabine Fuchs – form the Utrecht-based research group Omnes Pro Uno (OPU). The prize money will be divided evenly between them.
Curing genetic diseases
For ten years, research group OPU, winner in the Biomedical Sciences domain, has had the mission of seeking to find a cure for patients with a genetic disease before irreversible damage occurs in the body. Their research has led to groundbreaking world-first discoveries.
For example, researchers have succeeded in growing mini-organs from almost all tissues in the human body so that treatments can be tested and genetic errors corrected. The next ambitious step is for OPU to be the first in the world to correct the genetic cause of diseases directly in the bodies of patients via the bloodstream, before the disease has an opportunity adversely to affect the body. The approach will first be applied to children with a metabolism disease, but the idea could be more widely applicable.
Dr. Ruben van Boxtel: ‘Ultimately, we would like to prevent children from ending up in the Máxima in the first place. That is a long way off, but by taking part in this project we are involved early in the process in order to investigate the aspect of genetic safety. I am looking forward to this collaboration, and I also find the ethical aspect very interesting. It shows the value of joining forces at the Utrecht Science Park.’
Dr. Sabine Fuchs, OPU's principal investigator and pediatrician for metabolic diseases at UMC Utrecht, says: ‘By combining basic, clinical, social and ethical research in all phases of our work, our team offers a unique perspective on treatment to patients and society as a whole. Thanks to the Ammodo Science Award, our team can now use the unprecedented developments in genetic engineering ethically to develop innovative gene correction therapies to treat large group of patients with inherited diseases.’