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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Библиографические подробности
Главные авторы: 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.
Формат: Artigo
Язык:Inglês
Опубликовано: Public Library of Science 2012
Предметы:
Research Article
Online-ссылка: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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