Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE)

Laboratory presentation

INTEGRARE (Integrated Genetic Approaches and New Therapies for Rare Diseases, UMR_S951) is a research unit specializing in translational research and gene therapy of rare genetic diseases. The unit is mainly interested in myopathies, metabolic diseases, blood diseases or immune deficiency. Using recombinant viral vectors (AAV and lentiviral vectors) or CRISPR technologies, researchers design novel therapeutic approaches, defining the pharmacological parameters and the safety of the approaches, and study how to overcome the roadblocks notably to avoid immune responses to gene therapy.
Promising therapeutic candidates are developed towards the clinic through Genethon's therapeutic programs. Several clinical trials of gene therapy are currently underway.
INTEGRARE is a partnership between Généthon, (a private R et D organization created by AFM / Téléthon), Inserm and the Universities (University of Evry / Paris Saclay University, EPHE).