Sickle cell disease (SCD) is a serious blood disorder affecting millions of people, primarily those of African descent. A mutation in the gene that encodes a subunit of the oxygen-carrying molecule, hemoglobin, causes the disease. Scientists at St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard showed a precise genome editing approach, prime editing, can change mutated hemoglobin genes back to their normal form in SCD patient cells, which restores normal blood parameters after transplantation into mice. The findings were published in Nature Biomedical Engineering.
