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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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Pediatr Neurol
Prif Awduron: Li, Yedda, Sands, Mark S.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: 2014
Pynciau:
Mynediad Ar-lein: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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