Fabrice Lejeune, winner of the Rare Disease Foundation's call for screening projects, presents his innovative research project to treat certain rare genetic diseases, including mucovicidosis.
Click here to hear his testimony during his interview with Anne-Sophie Yribarren, the North-West manager of the Rare Disease Foundation.
For more information : Benhabiles H, Gonzalez-Hilarion S, Amand S, Bailly C, Prévotat A, Reix P, et al. (2017) Optimized approach for the identification of highly efficient correctors of nonsense mutations in human diseases. PLoS ONE 12(11): e0187930.