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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Detaylı Bibliyografya
Asıl Yazarlar: 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
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: American Society for Clinical Investigation 2013
Konular:
Research Article
Online Erişim: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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