Stem Cells Show Promise in Treating Sickle Cell
The Westside Gazette, News Report, K. Chandler, Posted: Jan 30, 2006
It was recently announced: preliminary scientific experiments using laboratory mice indicate that embryonic stem cells hold great promise for remedying the genetic mutation that causes sickle cell disease.
The studies, which were carried out at the University of California in San Francisco, used embryonic stem cells from mice that were harvested from days-old blastocysts with sickle cell. The DNA of the blastocysts was then manipulated so that the abnormal hemoglobin S was replaced by healthy hemoglobin A, which is merely the sickle cell trait minus the genetic markers that give rise to the full-blown expression of the debilitating disease.
The newly corrected hemoglobin A cells were then placed in a Petrie dish where they grew into hematopoietic stem cells, which are essentially cells found in adult bone marrow. Scientists hope that one day these cells will be able to be transplanted into sickle cell patients, eradicating the disease.
They urged caution however, as the newly-mined hemoglobin A stem cells have yet to be transplanted back into the laboratory mice either through a bone marrow transplant or cord blood transplant, so there is no guarantee they will effectively eliminate the genetic disorder or even be accepted by the host subject for that matter.
Increasing the odds of successfully transplanting normal hemoglobin A stem cells back into lab mice will entail suppressing the bone marrow of the mice through chemotherapy or radiation, biomedical researchers say, so that the healthy hemoglobin A stem cells have the greatest chance of being accepted rather than rejected.
According to J. Paul Scott, director of the Sickle Cell Comprehensive Program at Children’s Hospital of Wisconsin, there is good reason to hope that the medical breakthrough will be “translated in the next few years” into something that will benefit the “sickest patients.” But first, he cautioned, it must be proven conclusively that it works in laboratory mice and that it’s safe in the long haul.
Despite the fact that no human trials have been held so far, the implications for people suffering from this painful genetic disorder – which primarily affects the bones of the spine, legs, arms, chest and abdomen — are profound, possibly enabling them to one day be freed from the excruciatingly painful and incapacitating episodes that characterize the disease.
Currently over 2.5 million people in this country carry the sickle cell trait and more than 70,000 actually have the disease, which predominantly affects African Americans and people of Mediterranean descent.
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