It is the first time that an induced pluripotent stem (iPS) cell line has been created from a patient with a genetic illness. Like embryonic stem cells, iPS cells have the potential to develop into almost any of the body’s cell types and offer new disease insights.
Researchers made the iPS cells using viral vectors to introduce four genes into skin cells taken from two elderly patients with a mild form of ALS (also known as Lou Gehrig’s disease). This genetic reprogramming technique was first developed in 2006 in Japan. Although results are not yet reported in the peer-reviewed literature, posters at a stem-cell meeting in June described iPS cell lines from people with Alzheimer’s disease, Down’s syndrome, muscular dystrophy, and more.
Such cell lines could be most useful for diseases that are hardest to research. For example, in ALS, because the dying neurons reside within the spinal cord, they are nearly impossible to study in living patients, says Henderson. “We now have cells in culture that are genetically the same as in those with the disease.”
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