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. 2015 Apr 22;2:15012. doi: 10.1038/mtm.2015.12

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Copyright © 2015 American Society of Gene & Cell Therapy

This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/

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Long-term assessment of VCN, βAS3-globin mRNA, and HbAS3 expression in peripheral blood from secondary mice. (a) VCN analyzed in blood samples 11 weeks post-transplant. No differences in VCN were found between mice treated with the two different LV (P = 0.65, two-sample t-test). (b) Percentage of βAS3-globin mRNA per VCN from the samples shown in (a). Expression values were higher in the group of mice treated with Ank-R LV (P = 0.001, two-sample t-test). (c) The percentages of βAS3-globin mRNA per VCN, expressed in the primary and secondary mice at week 20 and 11 post-transplant, respectively, were analyzed for each LV (two-sample t-test). FB LV: primary mice, n = 9 and secondary, n = 18; for Ank-R LV-treated mice: primary, n = 8 and secondary, n = 21. The data shown in this graph at week 20 of the primary mice correspond to the average values shown in Supplementary Figure S5b; and the data shown at week 11 for the secondary mice are the same graphed in this panel in 4(b). (d) HPLC of blood samples from secondary recipients at week 11 post-transplant for hemoglobin tetramers analysis. The relative percentage of HbAS3 produced for each sample was calculated based on the sum total of areas under the curve for each of the primary hemoglobin peaks which included βmajor and βminor. Protein production was higher in the group of mice treated with Ank-R LV (P = 0.002, two-sample t-test). Bars represent average values and SD.