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Series GSE21343 Query DataSets for GSE21343
Status Public on Apr 16, 2010
Title Global gene expression profiles of mouse ESCs, disease-specific iPSCs, and gene-corrected iPSCs
Organism Mus musculus
Experiment type Expression profiling by array
Summary The use of patient-derived induced pluripotent stem (iPS) cells as treatment for genetic diseases entails genetic repair or transfer of genetic information as a prerequisite. We have chosen the murine model of tyrosinemia type 1 (fumarylacetoacetate hydrolase deficiency; FAH(-/-) mice) as a paradigm for hereditary metabolic liver disorders and evaluated fibroblast-derived FAH(-/-)-iPS cell lines as targets for gene correction. By aggregating FAH(-/-)-iPS cells with tetraploid embryos, we obtained FAH-/--iPS cell–derived mice, which exhibited the phenotype of the founding FAH(-/-)-mice. We then rescued the diseased phenotype by lentiviral transduction of FAH-cDNA and performed embryo aggregation with these gene-corrected FAHgc-iPS cells to obtain viable healthy mice. Our results demonstrate that iPS cell technology is a valid approach to establish mouse disease models directly from somatic cells bearing genetic defects. Furthermore, established iPS cell lines can be genetically manipulated without loss of pluripotency for treatment of genetic diseases.
Overall design 6 samples: 1 MEF, 1ESC, 4 iPSCs
Citation(s) 21765802
Submission date Apr 14, 2010
Last update date Jun 14, 2018
Contact name Boris Greber
Organization name Max Planck Institute for Molecular Biomedicine
Department Cell and Developmental Biology
Street address Röntgenstraße 20
City Münster
ZIP/Postal code 48149
Country Germany
Platforms (1)
GPL6885 Illumina MouseRef-8 v2.0 expression beadchip
Samples (6)
GSM533277 FAH MEFs
GSM533303 FAH iPSCs #4
GSM533309 FAH iPSCs #7
BioProject PRJNA126139

Download family Format
SOFT formatted family file(s) SOFTHelp
MINiML formatted family file(s) MINiMLHelp
Series Matrix File(s) TXTHelp

Supplementary file Size Download File type/resource
GSE21343_RAW.tar 4.2 Mb (http)(custom) TAR (of TXT)
Raw data provided as supplementary file
Processed data included within Sample table

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