Two research teams have focused on reducing the immune response directed against the vector or against the transgene product, which limits the efficacy of gene therapy and prevents its re-administration:
- Giuseppe Ronzitti’s team (Genethon) has developed a gene therapy approach using an AAV9-derived vector targeting muscles (and limiting addressing to the liver), a hepatic promoter and the GAA gene; it improves muscle strength in treated mice and induces immunotolerance thanks to low hepatic production of GAA;
- Priya Kishnani’s team has developed an effective immunosuppressive treatment combining bortezomib and rituximab administered prior to re-injection of a gene therapy product into mouse models of Pompe disease.
