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

Abstract 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 b...

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Bibliografiske detaljer

Principais autores:	Nicolina Cristina Sorrentino, Luca D'Orsi, Irene Sambri, Edoardo Nusco, Ciro Monaco, Carmine Spampanato, Elena Polishchuk, Paola Saccone, Elvira De Leonibus, Andrea Ballabio, Alessandro Fraldi
Format:	Artigo
Sprog:	Inglês
Udgivet:	Springer Nature 2013-04-01
Serier:	EMBO Molecular Medicine
Fag:	blood‐brain barrier CNS therapy lysosomal storage disorders MPS‐IIIA sulphamidase
Online adgang:	https://doi.org/10.1002/emmm.201202083
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