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Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I
Mucopolysaccharidosis type I (MPS I) is a lysosomal disease caused by α-l-iduronidase (IDUA) deficiency and accumulation of glycosaminoglycans (GAG). Lentiviral vector encoding correct IDUA cDNA could be used for treating MPS I. To optimize the lentiviral vector design, 9 constructs were designed by...
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| Publicado en: | Mol Genet Metab Rep |
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| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
Elsevier
2016
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4987501/ https://ncbi.nlm.nih.gov/pubmed/27556013 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2015.11.004 |
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