Article
Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector.
The Biochemical journal - 1 Jun 2005
Di Natale Paola, Di Domenico Carmela, Gargiulo Nadia, Castaldo Sigismondo, Gonzalez Y Reyero Enrico, Mithbaokar Pratibha, De Felice Mario, Follenzi Antonia, Naldini Luigi, Villani Guglielmo R D
Abstract excerpt
The Sanfilippo syndrome type B (mucopolysaccharidosis IIIB) is an autosomal recessive disorder due to mutations in the gene encoding NAGLU (alpha-N-acetylglucosaminidase), one of the enzymes required for the degradation of the GAG (glycosaminoglycan) heparan sulphate. No therapy exists for affected patients. We have shown previously the efficacy of lentiviral-NAGLU-mediated gene transfer in correcting in vitro...
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