BCL11A and Schnyder corneal dystrophy: An updated analysis of this study was extended to 10 SCD patients, showing that post-transcriptional gene knockdown of BCL11A was safe, associated with stable HbF induction and significant mitigation of vascular occlusive events: in six SCD patients with frequent vascular occlusive events before treatment, between zero and one events of vascular occlusion after gene therapy were reported; in three SCD patients treated with chronic transfusion regimens before treatment, transfusion independence was reached in 2/3 cases [53].