The erythroid-specific knockdown of BCL11A via an LV-encoded microRNA-adapted shRNA (e.g., BCH-BB694) has been demonstrated to robustly induce HbF in both in vitro and in vivo settings without adverse effects on HSPC function, thus providing preclinical support for clinical translation in sickle cell disease and β-thalassemia [68]. The gene discussed is BCL11A; the disease is sickle cell disease.