By reprogramming mouse cells into stem cells and purging them of a disease mutation, researchers have spawned disease-free animals.
Tobias Cantz at Hanover Medical School in Germany and his colleagues began by making induced pluripotent stem cells (iPSCs) from the cells of mice lacking the enzyme FAH, which are used as a model of some hereditary liver diseases. The authors then delivered a working version of the Fah gene to the iPSCs with a retrovirus. Finally, they used these cells to create embryos and generate mice with working copies of Fah and no signs of liver disease.
Human iPSCs might be similarly manipulated to rid them of mutations and then converted into working tissues for transplantation.
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Edited stem cells yield healthy mice. Nature 475, 426 (2011). https://doi.org/10.1038/475426b
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DOI: https://doi.org/10.1038/475426b
