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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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| Auteurs principaux: | , , , , , , , , , |
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| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
Portland Press Ltd.
2005
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| Sujets: | |
| Accès en ligne: | 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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