Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
Titel:
Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
Auteur:
Muñoz, Sergio Bertolin, Joan Jimenez, Veronica Jaén, Maria Luisa Garcia, Miquel Pujol, Anna Vilà, Laia Sacristan, Victor Barbon, Elena Ronzitti, Giuseppe El Andari, Jihad Tulalamba, Warut Pham, Quang Hong Ruberte, Jesus VandenDriessche, Thierry Chuah, Marinee K. Grimm, Dirk Mingozzi, Federico Bosch, Fatima