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. 2014 Jan 7;22(3):567–574. doi: 10.1038/mt.2013.188

Figure 2.

Figure 2

Long-term expression of therapeutic levels of R338L human FIX in hemophilia B mice. (a) Depiction of the lentiviral vector (pTK1340) from which the codon-optimized R338L human FIX is expressed under the control of the human α1-antitrypsin promoter (hAAT). (b–g) Levels of human FIX concentration (ng/ml) and clotting activity (percentage normal human FIX clotting activity as measured in vitro) in hemophilia B mice treated with escalating doses of integration-deficient lentiviral vectors (IDLVs): (b) 45 µg p24gag, (c) 65 µg p24gag, and (d) 200 and 250 µg p24gag , compared with mice treated with (e) integrase-competent lentiviral vectors (ICLVs): (e) 7 and 20 µg p24gag, (f) 54 µg p24gag, and (g) 65 µg p24gag. All samples were measured in duplicates. Data are presented as mean ± SEM. cPPT, central polypurine tract; LTR, long terminal repeat; PBS, phosphate-buffered saline; PPT, polypurine tract; WPRE, woodchuck hepatitis virus posttranscriptional regulatory element.