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    Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy

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

    Published 2024
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