Rescue of an In Vitro Neuron Phenotype Identified in Niemann-Pick Disease, Type C1 Induced Pluripotent Stem Cell-Derived Neurons by Modulating the WNT Pathway and Calcium Signaling

Niemann-Pick disease, type C1 (NPC1) is a familial disorder that has devastating consequences on postnatal development with multisystem effects, including neurodegeneration. There is no Food and Drug Administration-approved treatment option for NPC1; however, several potentially therapeutic compound...

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Detaylı Bibliyografya
Yayımlandı:Stem Cells Transl Med
Asıl Yazarlar: Efthymiou, Anastasia G., Steiner, Joe, Pavan, William J., Wincovitch, Stephen, Larson, Denise M., Porter, Forbes D., Rao, Mahendra S., Malik, Nasir
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: AlphaMed Press 2015
Konular:
Pluripotent Stem Cells
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC4339848/
https://ncbi.nlm.nih.gov/pubmed/25637190
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.5966/sctm.2014-0127
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