Study: Potential for reprogrammed skin cells to be used in sickle cell therapy

Cambridge, Mass. - Researchers have found that by reprogramming skin cells to become stem cells, it may be possible to treat sickle cell anemia, reports BBC News.

In their study, published recently in the journal Science, the researchers say they had some success with this therapy in treating mice that have human-type sickle cell anemia. The study notes that while reprogrammed skin cells - called induced pluripotent stem (IPS) cells - hold tremendous promise, they could cause dangerous side effects, and that more safety work is needed before trials involving humans can occur.

In order to manufacture the IPS cells, the researchers began with cells from the skin of the diseased mice, then used retroviruses to insert therapeutic genes into the DNA of these cells. The study notes that while this was effective, the retroviruses can randomly change DNA elsewhere in the body, which could potentially lead to complications such as cancer. BBC News quoted lead researcher Rudolf Jaenisch, of the Whitehead Institute for Biomedical Research in Cambridge, as saying, “We need a delivery system that doesn’t integrate itself into the genome. Retroviruses can disrupt genes that should not be disrupted or activate genes that should not be activated.”

Should this skin-cell therapy ever be used in humans, the study notes, there will be two advantages: First, skin cells are plentiful; second, the use of reprogrammed skin cells as stem cells skirts the ethics issues involved in other stem-cell-based treatments.