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◆ Molecular therapy. Nucleic acids2026-09-08

Sub-stoichiometric 5-methoxyuridine modification enables tunable immune evasion and protein expression from synthetic mRNAs.

Jonathan R Miles, Sarah Masterson, Eleanor Bellows, Fabio Fisher, Ritu Rani, Sara Marelli, Colin Hardman, Luigi Grassi, Luis Santos, Victoria James, Nigel P Mongan, Christopher J Hayes, Matthew Loose, Janet M Daly, James Button, George Thom, Rupert G Fray, Nathan Archer

原始摘要(英文原文)· Original abstract
mRNA therapeutics depend on nucleotide modification to evade innate immunity and sustain translation. Current clinical designs replace all uridine with N1-methylpseudouridine (m1Ψ). However, full substitution is costly and can perturb translational fidelity and RNA structure. Here, we show that sub-stoichiometric incorporation of 5-methoxyuridine (5moU) is sufficient to confer immune evasion while enhancing protein output. Substitution of 25% of uridines with 5moU supported translation comparable to fully m1Ψ-modified mRNA in some cell types, while 50% 5moU maximized expression across all systems tested, often outperforming 100% m1Ψ-modified mRNA. This effect was conserved in human primary cells and in vivo, where lipid nanoparticle-delivered mRNA encoding human IgG produced approximately 2-fold higher serum levels with 50% 5moU than with 100% m1Ψ. Transcriptomic profiling revealed reduced interferon-stimulated gene induction following delivery of 5moU-modified mRNA compared with unmodified and m1Ψ modified mRNA. Unlike m1Ψ, 5moU modification consistently suppressed innate responses to double-stranded RNA. Together, these data demonstrate that partial nucleotide substitution can outperform current mRNA modification strategies and expand the design space for therapeutic mRNAs.
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Sub-stoichiometric 5-methoxyuridine modification enables tunable immune evasion and protein expression from synthetic mRNAs. — 科研速览 Science Skim