Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy

Intravascular injection of certain adeno-associated virus vector serotypes can cross the blood–brain barrier to deliver a gene into the CNS. However, gene distribution has been much more limited within the brains of large animals compared to rodents, rendering this approach suboptimal for treatment...

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Bibliografiske detaljer
Udgivet i:Brain
Main Authors: Yoon, Sea Young, Hunter, Jacqueline E, Chawla, Sanjeev, Clarke, Dana L, Molony, Caitlyn, O’Donnell, Patricia A, Bagel, Jessica H, Kumar, Manoj, Poptani, Harish, Vite, Charles H, Wolfe, John H
Format: Artigo
Sprog:Inglês
Udgivet: Oxford University Press 2020
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Original Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7363495/
https://ncbi.nlm.nih.gov/pubmed/32671406
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/brain/awaa161
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