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Experimental Therapies in the Murine Model of Globoid Cell Leukodystrophy

Globoid cell leukodystrophy (GLD), also known as Krabbe disease, is a rapidly progressing childhood lysosomal storage disorder caused by a deficiency in galactocerebrosidase (GALC). GALC-deficiency leads to the accumulation of galactosylsphingosine (psychosine), a cytotoxic lipid especially damaging...

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Bibliografiske detaljer
Udgivet i:Pediatr Neurol
Main Authors: Li, Yedda, Sands, Mark S.
Format: Artigo
Sprog:Inglês
Udgivet: 2014
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4252788/
https://ncbi.nlm.nih.gov/pubmed/25240259
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.pediatrneurol.2014.08.003
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