ArticleThe Biochemical journal2002
Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transfer.
Article in The Biochemical journal, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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Who cites it
9 citing papers in PubMed, 20 citations in OpenAlex.
- Novel therapies for mucopolysaccharidosis type III.Journal of inherited metabolic disease · 2021Review
- Glycosaminoglycan storage disorders: a review.Biochemistry research international · 2012Article
- Secondary alterations of sphingolipid metabolism in lysosomal storage diseases.Neurochemical research · 2011Review
- Deregulated sphingolipid metabolism and membrane organization in neurodegenerative disorders.Molecular neurobiology · 2010Review
- A versatile viral system for expression and depletion of proteins in mammalian cells.PloS one · 2009Article
- Collagen alpha1(I) gene (COL1A1) is repressed by RFX family.The Journal of biological chemistry · 2005Article
- Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector.The Biochemical journal · 2005Article
- Newborn liver gene transfer by an HIV-2-based lentiviral vector.Gene therapy · 2005Article
- Feasibility of gene therapy in Gaucher disease using an adeno-associated virus vector.Journal of human genetics · 2004Article
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Mucopolysaccharidosis type IIIB (MPS IIIB; or Sanfilippo syndrome type B) is a lysosomal disease, due to glycosaminoglycan storage caused by mutations on the alpha-N-acetylglucosaminidase (NAGLU) gene. The disease is characterized by neurological dysfunction but relatively mild somatic manifestations. No effective treatment is available for affected patients. In the present study, we evaluated the role of a lentiviral vector as the transducing agent of NAGLU cDNA in MPS IIIB fibroblasts. The vector expressed high transduction efficiency and high levels of enzymic activity, 20-fold above normal levels, persisting for at least 2 months. PCR experiments confirmed the integration of the viral vector into the target genome. The NAGLU activity restored by virus infection was sufficient to normalize glycosaminoglycan accumulation, which is directly responsible for the disease phenotype. Metabolic labelling experiments on transduced fibroblasts exhibited, in the medium and in cellular lysates, polypeptide forms of 84 and 80 kDa respectively related to the precursor and mature forms of the enzyme. The enzyme secreted by transduced MPS IIIB fibroblasts was endocytosed in deficient cells by the mannose 6-phosphate system. Thus we show that lentiviral vectors may provide a therapeutic approach for the treatment of MPS IIIB disease.
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