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Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration

Preclinical studies have demonstrated that a single injection of an adeno-associated virus (AAV) vector into the cerebrospinal fluid (CSF) can achieve widespread gene transfer throughout the central nervous system. Successfully translating this approach to humans requires identifying factors that in...

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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Hinderer, Christian, Katz, Nathan, Dyer, Cecilia, Goode, Tamara, Johansson, Julia, Bell, Peter, Richman, Laura, Buza, Elizabeth, Wilson, James M.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2020
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7218226/
https://ncbi.nlm.nih.gov/pubmed/32420410
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.04.012
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