Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes

Nuphar Veiga, Meir Goldsmith, Yasmin Granot, Daniel Rosenblum, Niels Dammes, Ranit Kedmi, Srinivas Ramishetti and Dan Peer ()
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Nuphar Veiga: George S. Wise Faculty of Life Sciences
Meir Goldsmith: George S. Wise Faculty of Life Sciences
Yasmin Granot: George S. Wise Faculty of Life Sciences
Daniel Rosenblum: George S. Wise Faculty of Life Sciences
Niels Dammes: George S. Wise Faculty of Life Sciences
Ranit Kedmi: George S. Wise Faculty of Life Sciences
Srinivas Ramishetti: George S. Wise Faculty of Life Sciences
Dan Peer: George S. Wise Faculty of Life Sciences

Nature Communications, 2018, vol. 9, issue 1, 1-9

Abstract: Abstract Therapeutic alteration of gene expression in vivo can be achieved by delivering nucleic acids (e.g., mRNA, siRNA) using nanoparticles. Recent progress in modified messenger RNA (mmRNA) synthesis facilitated the development of lipid nanoparticles (LNPs) loaded with mmRNA as a promising tool for in vivo protein expression. Although progress have been made with mmRNA-LNPs based protein expression in hepatocytes, cell specificity is still a major challenge. Moreover, selective protein expression is essential for an improved therapeutic effect, due to the heterogeneous nature of diseases. Here, we present a precision protein expression strategy in Ly6c+ inflammatory leukocytes in inflammatory bowel disease (IBD) induced mice. We demonstrate a therapeutic effect in an IBD model by targeted expression of the interleukin 10 in Ly6c+ inflammatory leukocytes. A selective mmRNA expression strategy has tremendous therapeutic potential in IBD and can ultimately become a novel therapeutic modality in many other diseases.

Date: 2018
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DOI: 10.1038/s41467-018-06936-1

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