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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Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society for Clinical Investigation 2013
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Research Article
Accesso online: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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