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Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA
BACKGROUND: Mucopolysaccharidosis type IIIA (MPS IIIA) is the most common of the mucopolysaccharidoses. The disease is caused by a deficiency of the lysosomal enzyme sulphamidase and results in the storage of the glycosaminoglycan (GAG), heparan sulphate. MPS IIIA is characterised by widespread stor...
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| Glavni autori: | , , , , , , , , |
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| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
BioMed Central
2007
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| Teme: | |
| Online pristup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC1783652/ https://ncbi.nlm.nih.gov/pubmed/17227588 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1479-0556-5-1 |
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