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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Detalles Bibliográficos
Autores principales: Trilck, Michaela, Hübner, Rayk, Seibler, Philip, Klein, Christine, Rolfs, Arndt, Frech, Moritz J
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2013
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3848807/
https://www.ncbi.nlm.nih.gov/pubmed/24044630
http://dx.doi.org/10.1186/1750-1172-8-144

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