MOBILE, Ala. (WALA) - Gene therapy for sickle cell disease (SCD) is a cutting-edge treatment that targets the root cause of the disorder—a genetic mutation in the HBB gene, which leads to the ...

It comes from the copy of an abnormal sickle or the HBB gene and one copy of the normal HBB gene. The gene is an evolutionary response to malaria, where humans begin to develop an abnormal HBB ...