Inducing HbF by downmodulating BCL11A in hematopoietic stem cells by shRNA is currently explored as treatment of SCD.133 Preliminary results of this “transcriptome editing” approach in three patients with SCD who were followed at least 6 months after gene therapy showed a lineage-specific induction of HbF and an improvement of hemolysis.93 Here, BCL11A is linked to Schnyder corneal dystrophy.