CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neu...
CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder
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United States: Public Library of Science
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English
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United States: Public Library of Science
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A major challenge for the treatment of many central nervous system (CNS) disorders is the lack of convenient and effective methods for delivering biological agents to the brain. Mucopolysaccharidosis II (Hunter syndrome) is a rare inherited lysosomal storage disorder resulting from a deficiency of iduronate-2-sulfatase (I2S). I2S is a large, highly...
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Full title
CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder
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TN_cdi_plos_journals_1323091267
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_1323091267
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ISSN
1932-6203
E-ISSN
1932-6203
DOI
10.1371/journal.pone.0030341
