Welcome to the academic research of Genethon. Genethon is a leading clinical-stage R&D organization specialized in gene therapy for rare genetic diseases. Genethon was established 30 years ago by the French Muscular Dystrophy Association (AFM/Telethon) with the long-term goal of treating neuro-muscular disorders with gene therapies. In this process, our translational research has led to exciting advances and experience in a variety of models and in collaboration with many colleagues around the world. International gene therapy studies in primary immune deficiencies and hematological disorders have paved the way and demonstrated the possible efficacy and safety of gene therapy through ex vivo applications. The possibility to treat systemically with IV administration of rAAVs was developed in models of metabolic diseases and congenital myopathies and several clinical studies are currently ongoing in these diseases, enabling a perspective for the treatment of muscular dystrophies. Our current efforts are to better understand the molecular pathology of the diseases of interest to develop the most appropriate therapeutic strategies. We also study the mechanisms of immune responses to viral gene therapy vectors, the use of specific immunomodulators with limited toxicity and ways to generate less immunogenic vector platforms. At the technological level, new therapeutic strategies such as CRISPR genome editing approaches are showing promising results, for instance, in triplet disease disorders.
Our laboratories are regrouped as a research unit called INTEGRARE ("integrated genetic approaches for the treatment of rare genetic diseases", also identified as UMR_S951, a mixed research unit affiliated with Inserm the premier French public research institute on human health, and with the local University of Evry/Université Paris Saclay). INTEGRARE recruits talented post-doctoral fellows, PhD students, and interns. If you are interested in joining us, please consult our "Opportunities" section.
Situated within the Génopole biotechnology cluster of Evry, our laboratory has access to state-of-the art platforms including a flow/imaging facility, a functional exploration animal center and a newly-established Inserm accelerator of technological research. Evry is situated in the south of Paris, France and is accessible by car or by public transportation. Consult our “Tools and Facilities” section and map.
I wish you all a productive year 2020 !
Anne GALY, PhD
Director of INTEGRARE UMR_S951