Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector

The Sanfilippo syndrome type B (mucopolysaccharidosis IIIB) is an autosomal recessive disorder due to mutations in the gene encoding NAGLU (α-N-acetylglucosaminidase), one of the enzymes required for the degradation of the GAG (glycosaminoglycan) heparan sulphate. No therapy exists for affected pati...

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Hlavní autoři: Di Natale, Paola, Di Domenico, Carmela, Gargiulo, Nadia, Castaldo, Sigismondo, Reyero, Enrico Gonzalez Y, Mithbaokar, Pratibha, De Felice, Mario, Follenzi, Antonia, Naldini, Luigi, Villani, Guglielmo R. D.
Médium: Artigo
Jazyk:Inglês
Vydáno: Portland Press Ltd. 2005
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Research Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC1138972/
https://ncbi.nlm.nih.gov/pubmed/15649123
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1042/BJ20041702
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