Mechanism-Based Combination Treatment Dramatically Increases Therapeutic Efficacy in Murine Globoid Cell Leukodystrophy

Globoid cell leukodystrophy (GLD, Krabbe disease) is a lysosomal storage disease (LSD) caused by a deficiency in galactocerebrosidase (GALC) activity. In the absence of GALC activity, the cytotoxic lipid, galactosylsphingosine (psychosine), accumulates in the CNS and peripheral nervous system. Oligo...

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Publicat a:J Neurosci
Autors principals: Hawkins-Salsbury, Jacqueline A., Shea, Lauren, Jiang, Xuntian, Hunter, Daniel A., Guzman, A. Miguel, Reddy, Adarsh S., Qin, Elizabeth Y., Li, Yedda, Gray, Steven J., Ory, Daniel S., Sands, Mark S.
Format: Artigo
Idioma:Inglês
Publicat: Society for Neuroscience 2015
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Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4405559/
https://ncbi.nlm.nih.gov/pubmed/25904800
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1523/JNEUROSCI.4199-14.2015
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