CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder

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 i...

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Bibliografiske detaljer
Main Authors: Calias, Pericles, Papisov, Mikhail, Pan, Jing, Savioli, Nancy, Belov, Vasily, Huang, Yan, Lotterhand, Jason, Alessandrini, Mary, Liu, Nan, Fischman, Alan J., Powell, Jan L., Heartlein, Michael W.
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2012
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3261205/
https://ncbi.nlm.nih.gov/pubmed/22279584
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0030341
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