Overcoming the Blood-Brain Barrier with High-Dose Enzyme Replacement Therapy in Murine Mucopolysacch...
Overcoming the Blood-Brain Barrier with High-Dose Enzyme Replacement Therapy in Murine Mucopolysaccharidosis VII
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United States: National Academy of Sciences
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Language
English
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Publisher
United States: National Academy of Sciences
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Contents
Enzyme replacement therapy (ERT) effectively reverses storage in several lysosomal storage diseases. However, improvement in brain is limited by the blood-brain barrier except in the newborn period. In this study, we asked whether this barrier could be overcome by higher doses of enzyme than are used in conventional trials. We measured the distribu...
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Full title
Overcoming the Blood-Brain Barrier with High-Dose Enzyme Replacement Therapy in Murine Mucopolysaccharidosis VII
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TN_cdi_proquest_miscellaneous_68679983
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_68679983
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ISSN
0027-8424
E-ISSN
1091-6490
DOI
10.1073/pnas.0506892102
