Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE)
… Documents Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV … and lentiviral vectors) or CRISPR technologies, researchers design novel therapeutic approaches, defining the … therapy. Promising therapeutic candidates are developed towards the clinic through Genethon's therapeutic programs. …
Published on: