Skin cells reprogrammed for pluripotency can be used to treat anemia in a mouse model of the disease, reports a study published online in Science today (December 6). The researchers, led by linkurl:Rudolph Jaenisch;http://www.wi.mit.edu/research/faculty/jaenisch.html and Jacob Hanna at MIT, say the study provides proof of principle that linkurl:induced pluripotent stem cells;http://www.the-scientist.com/blog/display/53873/ (iPS cells) can be used to treat diseases. The scientists first created "humanized" mouse models for anemia by using gene knock-in techniques to replace mouse hemoglobin genes with human versions that cause the disease. Mice homozygous for the allele developed anemia symptoms and died within 18 months. They used four transcription factors -- Oct4, Sox2, Klf4 and c-Myc -- to generate lines of iPS cells from skin cells of the anemic mice. They then "corrected" these cells by replacing the diseased gene with its wild-type counterpart using homologous recombination techniques -- an approach that has been linkurl:used;http://www.ncbi.nlm.nih.gov/sites/entrez?orig_db=PubMed&db=PubMed&cmd=Search&term=Blood%5BJour%5D%20AND%20108%5Bvolume%5D%20AND%201183%5Bpage%5D%20AND%202006%5Bpdat%5D%20AND%20Wu%5Bauthor%5D with ES...
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