scholarly journals The Generation of Definitive Neural Stem Cells from PiggyBac Transposon-Induced Pluripotent Stem Cells Can Be Enhanced by Induction of the NOTCH Signaling Pathway

2013 ◽  
Vol 22 (3) ◽  
pp. 383-396 ◽  
Author(s):  
Ryan P. Salewski ◽  
Josef Buttigieg ◽  
Robert A. Mitchell ◽  
Derek van der Kooy ◽  
Andras Nagy ◽  
...  
Keyword(s):  
Stem Cells ◽  
Neural Stem Cells ◽  
Notch Signaling ◽  
Induced Pluripotent Stem Cells ◽  
Signaling Pathway ◽  
Pluripotent Stem Cells ◽  
Notch Signaling Pathway ◽  
Piggybac Transposon ◽  
Induced Pluripotent

scholarly journals Modeling CNS Involvement in Pompe Disease Using Neural Stem Cells Generated from Patient-Derived Induced Pluripotent Stem Cells

Cells ◽  
2020 ◽  
Vol 10 (1) ◽  
pp. 8
Author(s):  
Yu-Shan Cheng ◽  
Shu Yang ◽  
Junjie Hong ◽  
Rong Li ◽  
Jeanette Beers ◽  
...  
Keyword(s):  
Stem Cells ◽  
Neural Stem Cells ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Pompe Disease ◽  
Drug Efficacy ◽  
Cns Involvement ◽  
Glycogen Accumulation ◽  
Induced Pluripotent ◽  
Alpha Glucosidase

Pompe disease is a lysosomal storage disorder caused by autosomal recessive mutations in the acid alpha-glucosidase (GAA) gene. Acid alpha-glucosidase deficiency leads to abnormal glycogen accumulation in patient cells. Given the increasing evidence of central nervous system (CNS) involvement in classic infantile Pompe disease, we used neural stem cells, differentiated from patient induced pluripotent stem cells, to model the neuronal phenotype of Pompe disease. These Pompe neural stem cells exhibited disease-related phenotypes including glycogen accumulation, increased lysosomal staining, and secondary lipid buildup. These morphological phenotypes in patient neural stem cells provided a tool for drug efficacy evaluation. Two potential therapeutic agents, hydroxypropyl-β-cyclodextrin and δ-tocopherol, were tested along with recombinant human acid alpha-glucosidase (rhGAA) in this cell-based Pompe model. Treatment with rhGAA reduced LysoTracker staining in Pompe neural stem cells, indicating reduced lysosome size. Additionally, treatment of diseased neural stem cells with the combination of hydroxypropyl-β-cyclodextrin and δ-tocopherol significantly reduced the disease phenotypes. These results demonstrated patient-derived Pompe neural stem cells could be used as a model to study disease pathogenesis, to evaluate drug efficacy, and to screen compounds for drug discovery in the context of correcting CNS defects.

scholarly journals In vivo differentiation of induced pluripotent stem cells into neural stem cells by chimera formation

PLoS ONE ◽  
2017 ◽  
Vol 12 (1) ◽  
pp. e0170735 ◽  
Author(s):  
Hyun Woo Choi ◽  
Yean Ju Hong ◽  
Jong Soo Kim ◽  
Hyuk Song ◽  
Ssang Gu Cho ◽  
...  
Keyword(s):  
Stem Cells ◽  
Neural Stem Cells ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
In Vivo Differentiation ◽  
Induced Pluripotent

scholarly journals PI3K/AKT Signaling Pathway Is Essential for Survival of Induced Pluripotent Stem Cells

PLoS ONE ◽  
2016 ◽  
Vol 11 (5) ◽  
pp. e0154770 ◽  
Author(s):  
Amir M. Hossini ◽  
Annika S. Quast ◽  
Michael Plötz ◽  
Katharina Grauel ◽  
Tarik Exner ◽  
...  
Keyword(s):  
Stem Cells ◽  
Induced Pluripotent Stem Cells ◽  
Signaling Pathway ◽  
Pluripotent Stem Cells ◽  
Akt Signaling ◽  
Akt Signaling Pathway ◽  
Induced Pluripotent
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