NPR Reported 19 December: Scientists report progress using gene therapy to treat sickle cell disease, a common and devastating genetic blood disorder. New genetic technologies offer promise to treat it. Scientists are also renewed interest in older DNA techniques to help people with this common, devastating blood disorder.
One approach involves giving sickle cell patients' cells a new gene to compensate for the defective one, to make their bodies produce a healthy version of a protein called hemoglobin, and that's what red blood cells need to carry oxygen in the body. Another strategy is sort of similar to the CRISPR gene-editing approach that (a US patient) got; it involves using gene therapy to make cells produce a different kind of hemoglobin called fetal hemoglobin to make up for that defective hemoglobin.
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