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, … A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III Lucille … and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9 …
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