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 ...

It is caused by a mutation in the hemoglobin gene (HBB). CRISPR-Cas9 acts as molecular scissors that can precisely snip out parts of the HBB genome in living cells. To remedy sickle cells ...

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 ...