Bone Marrow Transplantation Augments the Effect of Brain- and Spinal Cord-Directed Adeno-Associated Virus 2/5 Gene Therapy by Altering Inflammation in the Murine Model of Globoid-Cell Leukodystrophy

Globoid-cell leukodystrophy (GLD) is an inherited demyelinating disease caused by the deficiency of the lysosomal enzyme galactosylceramidase (GALC). A previous study in the murine model of GLD (twitcher) demonstrated a dramatic synergy between CNS-directed adeno-associated virus 2/5 (AAV2/5) gene t...

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Hauptverfasser: Reddy, Adarsh S., Kim, Joong H., Hawkins-Salsbury, Jacqueline A., Macauley, Shannon L., Tracy, Elisabeth T., Vogler, Carole A., Han, Xialin, Song, Sheng-Kwei, Wozniak, David F., Fowler, Stephen C., Klein, Robyn S., Sands, Mark S.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Society for Neuroscience 2011
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Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3348856/
https://ncbi.nlm.nih.gov/pubmed/21734286
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1523/JNEUROSCI.1802-11.2011
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