Publications by authors named "Nathalie F Daniele"
Article Synopsis
- Glycogen storage disease type III (GSDIII) is a rare condition caused by a deficiency in the glycogen debranching enzyme, leading to liver issues and muscle weakness, with no current cure available.
- Previous research indicated that using two dual AAV (adeno-associated virus) vectors to deliver the GDE gene could effectively target both liver and muscle in a GSDIII mouse model.
- This study investigated the combination of rapamycin and AAV gene therapy, finding that the treatment improved outcomes by reducing immune response and enhancing the therapeutic effect, supporting further clinical applications.
