BCL11A and Schnyder corneal dystrophy: Exagamglogene autotemcel therapy (formerly known as CTX001) has been approved by the FDA for transfusion-dependent β-thalassemia and SCD; however, with reference to the example of elivaldogene autotemcel gene therapy consisting of autologous CD34+ cells leading to the development of hematological cancers in seven patients [101], for BCL11A erythroid enhancer editing, there may be a risk of secondary hematological malignancies in patients with transfusion-dependent thalassemia.