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Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy

Virginia Haurigot Mendonça, Miguel Garcia, Xavier Moll Sánchez, Martí Pumarola i Batlle, Jesús Ruberte, Sònia Añor Torres, Fàtima Bosch i Tubert, Albert Ribera Sánchez, Sara Marcó, Sandra Motas, Pilar Villacampa Alcubierre, Luca Maggioni, Xavier León, María Molas, Víctor Sánchez, Sergio Muñoz, Christian Leborgne, Federico Mingozzi

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Biochemistry, Genetics and Molecular Biology

Pharmacology, Toxicology and Pharmaceutical Science

Neuroscience