Article
Altered Differentiation Potential of Gaucher's Disease iPSC Neuronal Progenitors due to Wnt/β-Catenin Downregulation.
Stem cell reports - 12 Dec 2017
Awad Ola, Panicker Leelamma M, Deranieh Rania M, Srikanth Manasa P, Brown Robert A, Voit Antanina, Peesay Tejasvi, Park Tea Soon, Zambidis Elias T, Feldman Ricardo A
Abstract excerpt
Gaucher's disease (GD) is an autosomal recessive disorder caused by mutations in the GBA1 gene, which encodes acid β-glucocerebrosidase (GCase). Severe GBA1 mutations cause neuropathology that manifests soon after birth, suggesting that GCase deficiency interferes with neuronal development. We found that neuronopathic GD induced pluripotent stem cell (iPSC)-derived neuronal progenitor cells (NPCs) exhibit...
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