Generation of Healthy Mice from Gene-Corrected Disease-Specific Induced Pluripotent Stem Cells

2011

Creating Healthy Mice from Gene-Corrected Stem Cells

Sample size: 644 publication 15 minutes Evidence: high

Author Information

Author(s): Wu Guangming, Liu Na, Rittelmeyer Ina, Sharma Amar Deep, Sgodda Malte, Zaehres Holm, Bleidißel Martina, Greber Boris, Gentile Luca, Han Dong Wook, Rudolph Cornelia, Steinemann Doris, Schambach Axel, Ott Michael, Schöler Hans R., Cantz Tobias

Primary Institution: Max-Planck-Institute for Molecular Biomedicine, Münster, Germany

Hypothesis

Can gene-corrected induced pluripotent stem cells (iPS cells) be used to generate viable mice that exhibit normal liver function?

Conclusion

The study successfully generated healthy mice from gene-corrected iPS cells, demonstrating the potential of this approach for treating genetic diseases.

Supporting Evidence

The gene correction was validated functionally by the long-term survival of FAH-positive cells in the mice.
High efficiency of full-term development of up to 63.3% was achieved using gene-corrected iPS cells.
Both liver-specific and strong viral promoters successfully rescued the FAH-deficiency phenotypes in the derived mice.

Takeaway

Scientists made sick mice healthy by fixing their genes in special cells and then using those cells to create new mice.

Methodology

The researchers used fibroblast-derived iPS cells from a mouse model of tyrosinemia, corrected the genes using a lentiviral vector, and then used tetraploid embryo complementation to generate healthy mice.