Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

Virginia Haurigot, Sara Marcó, Albert Ribera Sanchez, Miguel Garcia Martinez, Albert Ruzo, Pilar Villacampa, Eduardo Ayuso Lopez, Sonia Añor Torres, Ana Maria Andaluz Martinez, Mercedes Pineda, Gemma García-Fructuoso, Maria Molas, Luca Maggioni, Sergio Muñoz, Sandra Motas, Jesus Ruberte Paris, Federico Mingozzi, Marti Pumarola Batlle, Maria Fatima Bosch Tubert

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187 Citations (Scopus)

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Keyphrases

Biochemistry, Genetics and Molecular Biology

Pharmacology, Toxicology and Pharmaceutical Science

Neuroscience