Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy

Globoid cell leukodystrophy (GLD, Krabbe disease), is an autosomal recessive, neurodegenerative disease caused by the deficiency of the lysosomal enzyme galactocerebrosidase (GALC). In the absence of GALC, the toxic metabolite psychosine accumulates in the brain and causes the death of the myelin-pr...

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Main Authors: Qin, Elizabeth Y., Hawkins-Salsbury, Jacqueline A., Jiang, Xuntian, Reddy, Adarsh S., Farber, Nuri B., Ory, Daniel S., Sands, Mark S.
Formato: Artigo
Idioma:Inglês
Publicado: 2012
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3444533/
https://ncbi.nlm.nih.gov/pubmed/22704480
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2012.05.021
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