Fragile X syndrome affects approximately 1 in 4 to 5,000 boys and 1 in 8,000 girls, or about 110,000 patients in Europe. It is now the leading inherited cause of intellectual disability and the leading known cause of autism spectrum disorders, and it does not yet have a cure.

The GETEX project focuses on the development of a new therapeutic approach to Fragile X syndrome, which proposes to compensate by gene therapy for the reduction of DGKk, an innovative therapeutic target and a potentially key actor in neuronal plasticity, whose synthesis is under the control of FMRP, the missing protein responsible for Fragile X syndrome.