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A first step to open the neuronal box of Gaucher Cells

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This work focuses on the differentiation and gene expression characterization of neural progenitor cells obtained from human induced pluripotent cells (hiPSCs) reprogrammed from type 3 GD (GD3) fibroblasts. GD3 patient fibroblasts (from an international cell bank) were cultured and reprogramed as previously described (https://doi.org/10.1016/j.scr.2019.101595). The resulting hiPSCs were differentiated into pre-neuronal cells and, at this stage, they were examined. The gene expression behavior of all neurogenesis genes (NES, MAP2 and OTX2) was similar but higher expression was observed in GD3 hiPSCs than in GD3 neural progenitor cells. With this work, we can conclude that, when working with hiPSCs in the process of creating disease-specific cell models it is most important to carry out a general gene expression characterization of the different cell lines involved in all stages.

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Abstract publicado em: https://cms.cloudguideapp.com/v2/backend/uploads/documents/689_abstract-b_31238917.pdf

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Human Genetics Tay Sachs disease induced Pluripotent Stem Cells Neurodegenerative Disease Lysosomal Disorders Modelos Celulares Gene Expression Gaucher Disease type 3 Doenças Genéticas

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