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Induced Pluripotent Stem Cell Modeling of Gaucher’s Disease: What Have We Learned?
Gaucher’s disease (GD) is the most frequently inherited lysosomal storage disease, presenting both visceral and neurologic symptoms. Mutations in acid β-glucocerebrosidase disrupt the sphingolipid catabolic pathway promoting glucosylceramide (GlcCer) accumulation in lysosomes. Current treatment opti...
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| Publicado no: | Int J Mol Sci |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
MDPI
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5412467/ https://ncbi.nlm.nih.gov/pubmed/28430167 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms18040888 |
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