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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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Detalhes bibliográficos
Main Authors: Huynh, Ha T., Grubb, Jeffrey H., Vogler, Carole, Sly, William S.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2012
Assuntos:
Acesso em linha: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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