Scientific Publication 

Dataset in Support of the Generation of Niemann-Pick Type C1

Author(s): Rolfs, Prof. Arndt, MD, Frech, Moritz J, PhD, Peter, Franziska, Trilck, Michaela, PhD, Rabenstein, Michael

Data presented in this article demonstrate the generation and characterization of two novel NPC1 patient-specific induced pluripotent stem cell lines. Read more!

Data presented in this article demonstrate the generation and characterization of two novel Niemann-Pick disease Type C1 (NPC1) patient-specific induced pluripotent stem cell (iPSC) lines. For reprogramming fibroblasts, carrying the novel homozygous mutation c.1180T>C and the prevalent homozygous mutation c.3182T>C, were used. Reprogramming into patient-specific iPSCs was induced by retroviral transduction of the transcription factors Sox2, Klf4, Oct4 and c-Myc, and confirmed according to their pluripotency.


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