Cell-based High-throughput Screen identifies GALC Enhancers as Potential Small Molecules Therapies for Krabbe Disease

Krabbe disease, also known as globoid-cell leukodystrophy (GLD), is a lysosomal storage disease (LSD) caused by the deficiency of the lysosomal enzyme β-galactocerebrosidase (GALC), resulting in severe neurological manifestations related to demyelination secondary to elevated galactosylsphingosine (...

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Xehetasun bibliografikoak
Argitaratua izan da:J Neurosci Res
Egile Nagusiak: Jang, Dae Song, Ye, Wenjuan, Guimei, Tian, Solomon, Melani, Southall, Noel, Hu, Xin, Marugan, Juan, Ferrer, Marc, Maegawa, Gustavo H.B.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 2016
Gaiak:
Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC5328637/
https://ncbi.nlm.nih.gov/pubmed/27638606
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jnr.23875
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