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Keyword: lentiviralvector

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  • Sickle Cell Disease Corrected In Human Models Using Stem Cell-based Gene Therapy

    12/27/2005 12:53:22 AM PST · by neverdem · 9 replies · 462+ views
    In a study to be published in the January 2006 issue of Nature Biotechnology, researchers led by a team of scientists at Memorial Sloan-Kettering Cancer Center have devised a novel strategy that uses stem cell-based gene therapy and RNA interference to genetically reverse sickle cell disease (SCD) in human cells. This research is the first to demonstrate a way to genetically correct this debilitating blood disease using RNA interference technology. To prevent the production of the abnormal hemoglobin that causes sickle cell disease, a viral vector was introduced in cell cultures of patients who have the disease. The vector carried...