Pluripotent Stem Cells for Disease Modeling and Drug Discovery in Niemann-Pick Type C1

The lysosomal storage disorders Niemann-Pick disease Type C1 (NPC1) and Type C2 (NPC2) are rare diseases caused by mutations in the NPC1 or NPC2 gene. Both NPC1 and NPC2 are proteins responsible for the exit of cholesterol from late endosomes and lysosomes (LE/LY). Consequently, mutations in one of...

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Detalles Bibliográficos
Autores principales: Völkner, Christin, Liedtke, Maik, Hermann, Andreas, Frech, Moritz J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7828283/
https://www.ncbi.nlm.nih.gov/pubmed/33445799
http://dx.doi.org/10.3390/ijms22020710

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7828283/
https://www.ncbi.nlm.nih.gov/pubmed/33445799
http://dx.doi.org/10.3390/ijms22020710

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