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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Detalles Bibliográficos
Autores principales: Yamazaki, Yoshiyuki, Hirai, Yukihiko, Miyake, Koichi, Shimada, Takashi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4076682/
https://www.ncbi.nlm.nih.gov/pubmed/24981028
http://dx.doi.org/10.1038/srep05506

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4076682/
https://www.ncbi.nlm.nih.gov/pubmed/24981028
http://dx.doi.org/10.1038/srep05506

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