Author(s): Rolfs, Prof. Arndt, MD, Frech, Moritz J, PhD, Peter, Franziska, Rost, Sebastian
As an analysis of affected cells is unfeasible in NPC1-patients, we developed an in vitro model system, based on cells derived from NPC1-patient specific iPSCs. Read more!
Niemann-Pick disease Type C1 (NPC1) is a rare progressive neurodegenerative disorder caused by mutations in the NPC1 gene. The pathological mechanisms, underlying NPC1 are not yet completely understood. Especially the contribution of glial cells and gliosis to the progression of NPC1, are controversially discussed. As an analysis of affected cells is unfeasible in NPC1-patients, we recently developed an in vitro model system, based on cells derived from NPC1-patient specific iPSCs.
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