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Dialing Back in Time the Entire Genome

In the last 3 years, a new technique for reprogramming adult cells has given scientists an easier and less controversial way to harness the power of embryonic-like stem cells to study human disease from its earliest beginnings in hopes of gleaning new insights into the root causes of disease and developing new therapies.But the reprogrammed cells, known as induced pluripotent stem (iPS) cells, are different from embryonic stem cells in their ability to model a human genetic disease, a new cell to cell comparison shows.

“This is the first example where we can clearly show induced pluripotent stem cells and embryonic stem cells behave differently in a disease model,” said co-senior author George Daley, Director of the Stem Cell Transplantation Program at Children’s Hospital Boston. Daley’s team has turned patient cells back into stem cells for a range of diseases.

In the new study in the May 7, Cell Stem Cell, the researchers made iPS cells from the skin cells of 3 patients with fragile X syndrome, the most common form of inherited mental retardation in boys. By almost every measure, virtually the entire genome was dialed back in time. The key exception was the disease-causing gene, which becomes inactivated to cause the disease, and did not get turned back on in the iPS cells. (HWM and PR Newswire)