Sialic Acid Deposition Impairs the Utility of AAV9, but Not Peptide-modified AAVs for Brain Gene Therapy in a Mouse Model of Lysosomal Storage Disease

Recombinant vector systems have been recently identified that when delivered systemically can transduce neurons, glia, and endothelia in the central nervous system (CNS), providing an opportunity to develop therapies for diseases affecting the brain without performing direct intracranial injections....

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Autori principali: Chen, Yong Hong, Claflin, Kristin, Geoghegan, James C, Davidson, Beverly L
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2012
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3392983/
https://ncbi.nlm.nih.gov/pubmed/22588273
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.100
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