Figure 4.
Lentiviral vector design with the cHS4 insulator.33 The SIN lentiviral backbone is flanked by a 1.2-kb cHS4 insulator (cHS4-I) element to replace the 398-bp U3 promoter and/or enhancer deletion. Upon proviral integration into host genome, the U3 region containing the cHS4 is copied over to the 5′LTR. The ‘insulated’ replacement gene contains the β-globin gene and β-globin LCR elements.
Reprinted with permission from
Puthenveetil G, Scholes J, Carbonell D, et al. Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector. Blood. 2004;104:3445–3453.
©American Society of Hematology.