Intrathecal Administration of AAV/GALC Vectors in 10-11 Day old Twitcher Mice Improves Survival and is Enhanced by Bone Marrow Transplant

Globoid cell Leukodystrophy (GLD), or Krabbe disease, is an autosomal recessive neurodegenerative disease caused by the deficiency of the lysosomal enzyme Galactocerebrosidase (GALC). Hematopoietic stem cell transplantation (HSCT) provides modest benefit in pre-symptomatic patients but well short of...

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Publicado en:J Neurosci Res
Autores principales: Karumuthil-Melethil, Subha, Marshall, Michael S., Heindel, Clifford, Jakubauskas, Benas, Bongarzone, Ernesto, Gray, Steven J.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2016
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC5028109/
https://ncbi.nlm.nih.gov/pubmed/27638599
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jnr.23882
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