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Manuscripts


Systemic Gene Silencing in Primary T Lymphocytes Using Targeted Lipid Nanoparticles
Ramishetti S, Kedmi R, Goldsmith M, Leonard F, Sprague AG, Godin B, Gozin M, Cullis PR, Dykxhoorn DM, Peer D. ACS Nano (2015) 9(7):6706-6716. Modulating T cell function by down-regulating specific genes using RNA interference (RNAi) holds tremendous potential in advancing targeted therapies in many immune-related disorders including cancer, inflammation, autoimmunity, and viral infections. Hematopoietic cells, in general, and primary T lymphocytes, in particular, are notoriou
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A modular platform for targeted RNAi therapeutics
Kedmi R, Veiga N, Ramishetti S, Goldsmith M, Rosenblum D, Dammes N, Hazan-Halevy I, Nahary L, Leviatan-Ben-Arye S, Harlev M, Behlke M, Benhar I, Lieberman J, Peer D. (2018) Previous studies have identified relevant genes and signaling pathways that are hampered in human disorders as potential candidates for therapeutics. Developing nucleic acid-based tools to manipulate gene expression, such as short interfering RNAs (siRNAs), opens up opportunities for personalized medicine.
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Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes
Veiga N, Goldsmith M, Granot Y, Rosenblum D, Dammes N, Kedmi R, Ramishetti S, Peer D. (2018) 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 e
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