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| 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.
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| |
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| Overall design |
6 samples: 1 MEF, 1ESC, 4 iPSCs
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| |
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| Citation(s) |
21765802 |
| |
| Submission date |
Apr 14, 2010 |
| Last update date |
Jun 14, 2018 |
| Contact name |
Boris Greber |
| E-mail(s) |
[email protected]
|
| 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 |
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| Samples (6)
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| Relations |
| BioProject |
PRJNA126139 |
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