Genetic base editing treats sickle cell disease in mice

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.

from Latest Science News -- ScienceDaily https://www.sciencedaily.com/releases/2021/06/210603171156.htm