Welcome to our website !


Our laboratories conduct translational research in the field of gene therapies for rare genetic diseases.


We have focused our attention on certain genetic disorders affecting skeletal muscle, metabolism, immune or blood systems, which have unmet medical needs and are amenable to gene therapy. We are developing treatments based on recombinant viral gene transfer vectors (rAAV and lentiviral vectors) or CRISPR genome editing tools. To better use these technologies we also explore how immune responses are ellicited against gene therapy vectors or gene-modified cells, aiming to promote tolerance to the treatments. Promising preclinical projects are developed into early phase clinical trials in partnership international networks of collaborators and clinicians. For more information, please consult our "Publications" list and "Links".


Our laboratories constitute the academic research of Genethona leading clinical-stage, non-profit R&D organization which was established by the French Muscular Dystrophy Association (AFM/Telethon)


At the academic level, our laboratories are regrouped as a unit called INTEGRARE ("integrated genetic approaches for the treatment of rare genetic diseases", also identified as UMR_S951, and which is 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 SaclayThe unit was recently renewed by Inserm. We are happy to continue to welcome highly-motivated students.


At the local level, we are situated within the Génopole biotechnology cluster of Evry, a vibrant campus with access to state-of-the art platforms including a flow/imaging facility and a functional exploration animal center.  Evry is situated in the south of Paris, France and is accessible by public transportation. 


If you are interested in joining us, please consult our "Opportunities" section. 


Anne GALY, PhD                                                                 

Director of INTEGRARE UMR_S951