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Abstract
The discovery several years ago that fibroblasts and other somatic cells from mice and humans can be reprogrammed to become inducible pluripotent stem (iPS) cells has created enthusiasm for their potential applications in regenerative medicine and for modeling human diseases. Two independent studies in this issue of the JCI provide evidence that iPS cells represent a promising source of hepatocytes for a wide range of applications, including cell transplantation, drug toxicity testing, patient-specific disease modeling, and even ex vivo gene therapy. But how far have we come?
Authors
Linda E. Greenbaum
Figure 2
Genetic diseases of the liver modeled in iPS cell–derived human hepatocytes.
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Rashid and colleagues investigated the potential to model inborn genetic diseases of the liver in human iPS cell–derived hepatocytes (19 ). Skin fibroblasts were obtained from patients with 5 inborn metabolic liver diseases: AAT, FH, GSD1a, hereditary tyrosinemia, and Crigler-Najjar syndrome. Fibroblasts were reprogrammed to become iPS cells by introducing retroviral vectors expressing KLF4, OCT4, SOX2, and c-myc. iPS cells from 3 of these diseases were then differentiated in culture to become hepatocytes and analyzed for defects specific to each genetic disease.
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ISSN: 0021-9738 (print), 1558-8238 (online)