Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE)
… Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy Elena Barbon, Giuseppe Ronzitti, Federico Mingozzi 03/01/24 Molecular Metabolism The myotubular … research Anna Buj-Bello 02/01/24 Neuromuscular Disorders A functional mini-GDE transgene corrects impairment in …Published on: Wed 20/09/2023 - 07:25