Since its creation, the Research Center in Myology has been actively involved in the evolution of scientific and technological knowledges enabling to design original gene therapy tools, targeting rare genetic diseases that have no treatment so fare. The application of these new therapeutic strategies in humans requires crucial steps of preliminary validations on cellular and animal models. Today, innovative methods of video-microscopy imaging enable fast and high-speed preclinical validation of the efficacy of these new gene therapy tools. The beneficial effect of therapeutic agents is thus monitored automatically in real time on living cells, relevant models for these human diseases, significantly reducing the number of animals used in biomedical research. The innovative system of cell imaging that we propose to implement, accelerates the preclinical progress of gene therapy, towards the next development of clinical trials in humans.