Genetic base editing treats sickle cell disease in mice

(Broad Institute of MIT and Harvard) Sickle cell disease leads to chronic pain, organ failure, and early death in patients worldwide. A team led by Broad Institute of MIT and Harvard and St. Jude Children's Research Hospital 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.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news