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AAV9-mediated central nervous system–targeted gene delivery via cisterna magna route in mice

Current barriers to the use of adeno-associated virus serotype 9 (AAV9) in clinical trials for treating neurological disorders are its high expression in many off-target tissues such as liver and heart, and lack of cell specificity within the central nervous system (CNS) when using ubiquitous promot...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Lukashchuk, Vera, Lewis, Katherine E, Coldicott, Ian, Grierson, Andrew J, Azzouz, Mimoun
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4756767/
https://ncbi.nlm.nih.gov/pubmed/26942208
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.55
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