Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

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

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124 Citations (Scopus)

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Medicine & Life Sciences