BCL11A and Schnyder corneal dystrophy: Over the past three decades, research in gene therapy has evolved significantly, leading to FDA approval of two different gene therapies in 2023 for SCD patients > 12 years old with recurrent vaso-occlusive crises: exagamglogene autotemcel (CasgevyTM) employs CRISPR/Cas9 gene editing technology to downregulate BCL11A, thus inhibiting transition from HbF to HbA.