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. 2020 Apr 27;10:7052. doi: 10.1038/s41598-020-64017-0

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© The Author(s) 2020

Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.

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In vivo delivery and analysis of modified mRNA encoding human AAT into the NSG-PiZ mouse model of AATD. (a) Whole liver was sectioned and in situ hybridization was used to show global distribution of mRNA in the liver following intravenous delivery by tail-vein injection. (b) Biochemical analysis did not reveal any sign of cellular stress or damage caused by mRNA delivery. (c) Most of the mRNA can be detected by in situ hybridization in the liver sinusoids 2 hours after injection. The mRNA subsequently relocated into hepatocytes by 24 and 48 hours after injection. Hepatocytes with and without globules contained mRNA. (d) A large amount of mRNA could be detected by qPCR in analysed liver tissue. (e) Elastase activity assay showing inhibition in serum from animals receiving AAT mRNA and PBS treated animals.