Figure 10.
Sickle cell cures (Tisdale et al., 2020). Allogeneic stem cell transplantation is an established curative strategy that uses bone marrow stem cells from an immunlogically matched normal or sickle trait donor (left image). In new and very promising approaches currently being developed, the patient’s own bone marrow cells are modified either with the addition of a β-globin gene that codes for a polymerization inhibitory β globin or by using Crispr-Cas technology to edit the β6 locus or reactivate HbF synthesis.