Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

For most lysosomal storage diseases (LSDs) affecting the CNS, there is currently no cure. The BBB, which limits the bioavailability of drugs administered systemically, and the short half-life of lysosomal enzymes, hamper the development of effective therapies. Mucopolysaccharidosis type IIIA (MPS II...

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Detalhes bibliográficos
Main Authors: Haurigot, Virginia, Marcó, Sara, Ribera, Albert, Garcia, Miguel, Ruzo, Albert, Villacampa, Pilar, Ayuso, Eduard, Añor, Sònia, Andaluz, Anna, Pineda, Mercedes, García-Fructuoso, Gemma, Molas, Maria, Maggioni, Luca, Muñoz, Sergio, Motas, Sandra, Ruberte, Jesús, Mingozzi, Federico, Pumarola, Martí, Bosch, Fatima
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2013
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3726158/
https://ncbi.nlm.nih.gov/pubmed/23863627
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI66778
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