Pluripotent cells treat anemia?

Written byAlla Katsnelson

| 1 min read

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 cells. According to the paper, it worked comparably well in iPS cells. In the final step, they transplanted blood cells differentiated from the corrected iPS cells into three anemic mice. Using PCR, they showed that the DNA of the treated mice carried both the diseased and the corrected genes; a range of blood tests demonstrated that disease symptoms had improved to a state "comparable to control mice." But the researchers warn that major issues with the technique remain to be addressed before it can be used in treatment. One of the transcription factors they used in reprogramming iPS cells is believed to linkurl:cause tumors,;http://www.the-scientist.com/blog/display/53946/ and using retroviral vectors may also cause cancer.

Pluripotent cells treat anemia?

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