We recently demonstrated gene therapy using a clinically applicable lentiviral vector driven by a cellular promoter, EFS, could promote red blood cell production and normal hematopoiesis in a mouse DBA model with RPS19 deficiency and human RPS19-deficient CD34+ HSPCs, with a low risk of mutagenesis and a highly polyclonal insertion site pattern [114]. Here, RPS19 is linked to Diamond-Blackfan anemia.