A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA

Mucopolysaccharidoses type IIIA (MPS-IIIA) is a neurodegenerative lysosomal storage disorder (LSD) caused by inherited defects of the sulphamidase gene. Here, we used a systemic gene transfer approach to demonstrate the therapeutic efficacy of a chimeric sulphamidase, which was engineered by adding...

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Bibliographische Detailangaben
Hauptverfasser: Sorrentino, Nicolina Cristina, D'Orsi, Luca, Sambri, Irene, Nusco, Edoardo, Monaco, Ciro, Spampanato, Carmine, Polishchuk, Elena, Saccone, Paola, De Leonibus, Elvira, Ballabio, Andrea, Fraldi, Alessandro
Format: Artigo
Sprache:Inglês
Veröffentlicht: WILEY-VCH Verlag 2013
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Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3662312/
https://ncbi.nlm.nih.gov/pubmed/23568409
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/emmm.201202083
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