Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal
大型动物溶酶体贮积症 AAV CNS 基因治疗的持久性和安全性的七年随访
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2021.09.017
Sara Marcó, Virginia Haurigot, Maria Luisa Jaén, Albert Ribera, Víctor Sánchez, Maria Molas, Miguel Garcia, Xavier León, Carles Roca, Xavier Sánchez, Joan Bertolin, Jennifer Pérez, Gemma Elias, Marc Navarro, Ana Carretero, Martí Pumarola, Anna Andaluz, Yvonne Espada, Sonia Añor, Fatima Bosch