Genetic base editing treats sickle cell disease in mice

Grantee News
June 4, 2021

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. Read more from the Broad Institute.