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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Библиографические подробности
Главные авторы: 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
Формат: Artigo
Язык:Inglês
Опубликовано: American Society for Clinical Investigation 2013
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Research Article
Online-ссылка: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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