BCL11A and sickle cell disease: In 2023, four more gene therapies were approved: Vyjuvek for epidermolysis bullosa [16]; Lyfgenia, which uses a lentiviral vector encoding the antisickling hemoglobin HbAT87Q, for sickle cell anemia [17]; Casgevy, which employs CRISPR-Cas9, the first gene editing therapy, to target the BCL11A erythroid-specific enhancer [18], also for sickle cell anemia; and Elevidys, which is used for Duchenne muscular dystrophy (DMD) [19], a genetic muscular degenerative disease affecting approximately 1 in 5,000 males.