BCL11A and Schnyder corneal dystrophy: Approaches that have shown early clinical promise to treat TDT and SCD include addition of a normal β-globin coding sequence by lentiviral vectors34 and induction of fetal hemoglobin (HbF) by Cas9-mediated disruption3,5 or shRNA-mediated suppression4 of BCL11A. Furthermore, the latter approach that reactivating the γ-globin gene by erythroid-specific knockdown of BCL11A is in early clinical development and achieved preliminary therapeutic progress3–5.