Pluripotent cells treat anemia?

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?

The Scientist ARCHIVES

Become a Member of

Meet the Author

Alla Katsnelson

Related Research Resources

Study Confirms Safety of Genetically Modified T Cells

Research Resources

Podcasts

Webinars

Videos

Infographics

eBooks

Meet Aunty and Tackle Protein Stability Questions in Research and Development

Detecting Residual Cell Line-Derived DNA with Droplet Digital PCR

Making Real-Time PCR More Straightforward

Characterizing Immune Memory to COVID-19 Vaccination

Products

Product News

Biotium Launches New Phalloidin Conjugates with Extended F-actin Staining Stability for Greater Imaging Flexibility

Latest AI software simplifies image analysis and speeds up insights for scientists

BioSkryb Genomics and Tecan introduce a single-cell multiomics workflow for sequencing-ready libraries in under ten hours

Agilent BioTek Cytation C10 Confocal Imaging Reader