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Niemann-Pick type C1 patient-specific induced pluripotent stem cells display disease specific hallmarks
BACKGROUND: Niemann-Pick type C1 disease (NPC1) is a rare progressive neurodegenerative disorder caused by mutations in the NPC1 gene. In this lysosomal storage disorder the intracellular transport and sequestration of several lipids like cholesterol is severely impaired, resulting in an accumulatio...
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| Main Authors: | , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
BioMed Central
2013
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3848807/ https://ncbi.nlm.nih.gov/pubmed/24044630 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-144 |
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