Figure 1.

Genetic strategies for sickle cell disease. Anti-sickling protein coding gene addition, fetal globin induction via knocking-down/silencing of repressors of γ-globin gene, and sickle mutation correction with genome engineering tools, particularly CRISPR/Cas9, are the main genetic approaches for sickle cell disease. However, low efficient gene transfer methods, editing rates, and safety issues are critical issues that needs to be addressed before starting clinical trials.