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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 no: | J Neurosci Res |
|---|---|
| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2016
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| Assuntos: | |
| Acesso em linha: | 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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