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. 2015 Jun 30;23(9):1456–1464. doi: 10.1038/mt.2015.103

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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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Engineered, unmodified Epo mRNA can elicit systemic physiological responses in swine and nonhuman primates. (a) Intravenous mRNA injection into pigs gives rise to high serum erythropoietin (EPO) levels as well as to substantial physiological effects. Animals received 1.3 mg of lipid nanoparticle (LNP) encapsulated porcine Epo mRNA (0.065 mg/kg) on day 0 and were analyzed for EPO levels and hematological parameters at various times. Protein levels before treatment were below the limit of detection of the assay. (b) Intravenous mRNA injection into macaques increases EPO levels, reticulocyte numbers as well as the hematocrit. Animals received 100 μg of LNP encapsulated M. fascicularis Epo mRNA (0.037 mg/kg) and were analyzed for EPO levels and hematological parameters before and at various times after treatment. Distinct symbols are assigned to individual animals. Pre, prevalue before treatment, n = 3 for pig experiment, n = 4 for macaque study.