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. 2017 Apr 20;25(8):1805–1814. doi: 10.1016/j.ymthe.2017.04.002

Figure 3.

Figure 3

Enforced Expression of RPS19 Derived from the EFS Promoter Is Sufficient to Rescue the Acute DBA Phenotype In Vivo

Enforced expression of RPS19 results in short-term rescue of the hematological defect in RPS19-deficient mice. (A) Experimental strategy to validate the short-term therapeutic potential of EFS-RPS19 and LCR-EFS-RPS19 vectors. (B) Transduction efficiency is shown. (C and D) GFP reconstitution and donor reconstitution are shown. (E–G) Erythrocyte number (E), hemoglobin concentration (F), and mean corpuscular value (G) (n = 20–21). Error bars represent the SD. ***p < 0.001. MCV, mean corpuscular value; Wt, wild-type.