A clinical study (NCT03653247) is assessing the safety, tolerability, and efficacy of the transplantation in Sickle Cell Disease patients of autologous HSPCs electroporated ex vivo with ZFN mRNAs targeting the B-cell lymphoma/leukemia 11A (BCL11A) locus, which plays a role in the fetal to adult erythropoiesis transition [239]. Here, BCL11A is linked to sickle cell disease.