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Gene therapy scientists move closer to ‘cure’ for sickle cell

ITN News Editor
By ITN News Editor March 12, 2019 12:28

Gene therapy scientists move closer to ‘cure’ for sickle cell

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.Researchers at the National Institutes of Health 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”.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.Collins says the disorder comes essentially from a “misspelling” in the gene for one of the proteins.

ITN News Editor
By ITN News Editor March 12, 2019 12:28

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