Our goals

Nonsense mutations cause about 10% of cases of genetic diseases. This percentage can be very variable from one genetic disease to another. For example, it is 8-12% in the case of cystic fibrosis or Duchenne muscular dystrophy but can reach 60% in the case of Rett Syndrome. The consequence of a nonsense mutation is the introduction of a premature termination codon which stops the synthesis of the protein leading to the absence of a function in the cell. GT aims to develop molecules to re-express the function of genes carrying nonsense mutations in order to provide a therapeutic solution.