Dataset Information


Homo sapiens

ABSTRACT: Global gene expression analysis of human iPSC-derived Neural Stem Cells (NSC) from LCA patients and unaffected persons

ORGANISM(S): Homo sapiens  



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Human induced pluripotent stem cells as a tool to model a form of Leber congenital amaurosis.

Lustremant Céline C   Habeler Walter W   Plancheron Alexandra A   Goureau Olivier O   Grenot Lydie L   de la Grange Pierre P   Audo Isabelle I   Nandrot Emeline F EF   Monville Christelle C  

Cellular reprogramming 20130510 3

Our purpose was to investigate genes and molecular mechanisms involved in patients with Leber congenital amaurosis (LCA) and to model this type of LCA for drug screening. Fibroblasts from two unrelated clinically identified patients with a yet undetermined gene mutation were reprogrammed to pluripotency by retroviral transduction. These human induced pluripotent stem cells (hiPSCs) were differentiated into neural stem cells (NSCs) that mimicked the neural tube stage and retinal pigmented epithel  ...[more]

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