Scientists from Johns Hopkins Medicine and eight other institutions in the United States, Africa and Europe say they have identified a potential new gene target that could be edited to treat sickle cell disease, an inherited blood disorder marked by sickle-shaped red blood cells that cause intense pain and shorten lifespans.
The potential target, the FLT1 gene, contributes to the production of a protein, fetal hemoglobin, whose presence is already known to improve the lifespan of people with sickle cell disease.