Abstract
Fragile X syndrome is a frequent form of genetic autism and intellectual disabilty. It has also become an prime example of translational reseach for neurodevelopmental disorders that are individually rare, but collectively frequent. Despite 30 years of intense reseach on the disorder, no targeted treatment has been approved. Part of the reason is that FMRP, the protein missing in fragile X syndrome, is involved in many cellular functions. In order to test novel drugs, we have therefore decided to join forces with Kantify, a drug development AI-company in Brussels (www.kantify.com). The company succesfully specializes in the development of novel drugs for rare diseases. We have provided our experimental data to help their software to predict novel drugs for the treatment of fragile X syndrome. From the list, we have selected three drugs that are already in use for other diseases, but have never been used to treat fragile X syndrome. The advantage of such ''repurposed drugs'' is that once these have been proven to be effective for treating symptoms, they are one of the fastest and most effective ways to get treatment to patients. We aim to test these novel drugs on our validated fragile X mouse model with the use of our sophisticated live mouse tracker system. This state-of-the-art system can measure fully automatic both individual and group behaviors of four mice over a 24h recording, to determine the efficiency of the treatments. It replaces the costly and time-consuming test batteries typically used in preclinical testing.
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