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, … 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 …
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