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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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| Publicado no: | J Neurosci Res |
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| Main Authors: | , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2016
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| Assuntos: | |
| Acesso em linha: | 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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