ArticleThe Biochemical journal2005
Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector.
Article in The Biochemical journal, 2005. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed, 60 citations in OpenAlex.
- Article
- Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.Biomolecules · 2021Review
- Novel therapies for mucopolysaccharidosis type III.Journal of inherited metabolic disease · 2021Review
- Dosing and Re-Administration of Lentiviral Vector forMolecular therapy. Methods & clinical development · 2020Article
- Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management.International journal of molecular sciences · 2020Review
- Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases.Frontiers in molecular biosciences · 2020Review
- Destabilizing Domains Enable Long-Term and Inert Regulation of GDNF Expression in the Brain.Molecular therapy. Methods & clinical development · 2018Article
- A model of mucopolysaccharidosis type IIIB in pigs.Biology open · 2018Article
- A novel mutation in the NAGLU gene associated with Sanfilippo syndrome type B (mucopolysaccharidosis III B).Clinical case reports · 2018Article
- The Murine Model of Mucopolysaccharidosis IIIB Develops Cardiopathies over Time Leading to Heart Failure.PloS one · 2015Article
- Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.Gene therapy · 2013Article
- Unfolded protein response is not activated in the mucopolysaccharidoses but protein disulfide isomerase 5 is deregulated.Journal of inherited metabolic disease · 2012Article
- Glycosaminoglycan storage disorders: a review.Biochemistry research international · 2012Article
- Activation of stress kinases in the brain of mucopolysaccharidosis IIIB mice.Journal of neuroscience research · 2011Article
- Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Serum MIP-1 alpha level: a biomarker for the follow-up of lentiviral therapy in mucopolysaccharidosis IIIB mice.Journal of inherited metabolic disease · 2010Article
- Sanfilippo syndrome type B, a lysosomal storage disease, is also a tauopathy.Proceedings of the National Academy of Sciences of the United States of America · 2009Article
- Gene therapy approaches for stem cell protection.Gene therapy · 2008Review
- Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA.Genetic vaccines and therapy · 2007Article
- Real-time quantitative PCR for the design of lentiviral vector analytical assays.Gene therapy · 2005Review
Corrections and comments
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Authors and funding
10 authors at 4 institutions in 1 country.
Funding
Abstract
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 the defect on fibroblasts of patients. In the present study, we tested the therapy in vivo on a knockout mouse model using intravenous injections. Mice (8-10 weeks old) were injected with one of the lentiviral doses through the tail vein and analysed 1 month after treatment. A single injection of lentiviral-NAGLU vector resulted in transgene expression in liver, spleen, lung and heart of treated mice, with the highest level reached in liver and spleen. Expression of 1% normal NAGLU activity in liver resulted in a 77% decrease in the GAG content; more remarkably, an expression of 0.16% normal activity in lung was capable of decreasing the GAG level by 29%. Long-term (6 months) follow up of the gene therapy revealed that the viral genome integration persisted in the target tissues, although the real-time PCR analysis showed a decrease in the vector DNA content with time. Interestingly, the decrease in GAG levels was maintained in liver, spleen, lung and heart of treated mice. These results show the promising potential and the limitations of lentiviral-NAGLU vector to deliver the human NAGLU gene in vivo.
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Registered trials
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