Solution to 50-year-old mystery could lead to gene therapy for common blood disorders

In a landmark study that could lead to new therapies for sickle cell anemia and other blood disorders, researchers have used CRISPR gene editing to introduce beneficial natural mutations into blood cells to boost their production of fetal hemoglobin. The research solves a 50-year-old mystery about how these mutations — which are naturally carried by a small percentage of people — operate and alter the expression of human genes.