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Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-l-iduronidase Fusion Gene Product
Mucopolysaccharidosis type I (Hurler syndrome) is caused by a deficiency of the enzyme α-l-iduronidase (IDUA), and is characterized by widespread lysosomal glycosaminoglycan (GAG) accumulation. Successful treatment of central nervous system (CNS) diseases is limited by the presence of the blood–brai...
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| Main Authors: | , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2008
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2574880/ https://ncbi.nlm.nih.gov/pubmed/18523448 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.119 |
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