Sickle Cell Disease is a complex and debilitating genetic blood disorder that primarily affects people of African, Mediterranean, Middle Eastern, and Indian ancestry.It is caused by a mutation in the ...

Beti-cel uses a self-inactivating lentiviral vector to deliver a functional copy of the HBB gene into patients' blood stem cells, allowing the cells to produce normal beta-globin protein and to ...

View Full Profile. Learn about our Editorial Policies. Today, the cause of sickle cell disease is well understood to be a point mutation in a hemoglobin gene called HBB that makes red blood cells grow ...