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Targeted gene transfer into ependymal cells through intraventricular injection of AAV1 vector and long-term enzyme replacement via the CSF

Enzyme replacement via the cerebrospinal fluid (CSF) has been shown to ameliorate neurological symptoms in model animals with neuropathic metabolic disorders. Gene therapy via the CSF offers a means to achieve a long-term sustainable supply of therapeutic proteins within the central nervous system (...

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מידע ביבליוגרפי
Main Authors: Yamazaki, Yoshiyuki, Hirai, Yukihiko, Miyake, Koichi, Shimada, Takashi
פורמט: Artigo
שפה:Inglês
יצא לאור: Nature Publishing Group 2014
נושאים:
גישה מקוונת:https://ncbi.nlm.nih.gov/pmc/articles/PMC4076682/
https://ncbi.nlm.nih.gov/pubmed/24981028
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep05506
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