• Genetic base editing treats sickle cell disease in mice

    13 days ago - By NIBIB / NIH

    Genetic base editing treats sickle cell disease in mice
    Grantee News thomas.johnson
    Fri, 06/04/2021 - 08:14
    Sickle cell disease leads to chronic pain, organ failure, and early death in patients worldwide. A team has demonstrated a gene editing approach that efficiently corrects the mutation underlying SCD in patient blood stem cells and in mice. This treatment rescued disease symptoms in animal models, enabling long-lasting production of healthy blood cells, and could inspire a therapeutic strategy for SCD.
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