Sickle cell may get solution soon – scientists.

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Scientists in the U.S. have unveiled results of a small clinical trial that could mean an effective “cure” for sickle cell anemia, the painful and debilitating disease that inflicts many millions of people across the globe, mostly of African heritage and including some 100,000 African Americans in the U.S.



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Researchers at the National Institutes of Health (NIH) say they have used gene therapy techniques to add a “corrected” gene for healthy red blood cells into the bodies of nine test patients, replacing their diseased red blood cells caused by sickle cell anemia and effectively ridding them of signs of the disease.

NIH Director Francis Collins described the trial results as seemingly “spectacular”.

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“When you look at their blood counts and their blood smears, it looks like they don’t have it anymore,” Collins said on Monday (March 11) from his office at the NIH in Bethesda, Maryland, just outside Washington, D.C.

Sickle cell disease is an inherited blood disorder that causes protein crystals to form inside red blood cells, changing their shape from a flat disk into a crescent or sickle shape that then clogs up the small blood vessels and results in terrible episodes of pain and organ damage.

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But they believe the basic premise of introducing a corrected gene into the body holds promise for Africa provided a simpler, cheaper and less toxic delivery system than bone marrow transplant and the accompanying chemotherapy can be found.

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