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Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII
Enzyme replacement therapy has been used successfully in many lysosomal storage diseases. However, correction of brain storage has been limited by the inability of infused enzyme to cross the blood–brain barrier (BBB). We recently reported that PerT-GUS, a form of β-glucuronidase (GUS) chemically mo...
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| Autors principals: | , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
National Academy of Sciences
2012
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3479451/ https://ncbi.nlm.nih.gov/pubmed/23027951 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1214779109 |
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