Article

Modified self-amplifying RNA mediates robust and prolonged gene expression in the mouse and ex vivo human brain

2025-10-31

Abstract excerpt

<h4>ABSTRACT</h4> In self-amplifying ribonucleic acid (saRNA), substitution of cytidine with 5-hydroxymethylcytidine (hm5C) reduces innate immune responses and prolongs protein expression. Administration routes to date for hm5C modified saRNA encapsulated within lipid nanoparticle (LNPs) include intramuscular, as a potent low dose vaccine, but expression levels, patterns, and cell tropism in other key organs are...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 73f5efd4-5667-58ab-b7d5-309fbb149d39
DOI: 10.1101/2025.10.30.685635

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Modified self-amplifying RNA mediates robust and prolonged gene expression in the mouse and ex vivo human brainDOI 10.1101/2025.10.30.685635Read

Albumin-binding dendrimer-conjugated siRNA enables safe and effective gene silencing throughout the central nervous systemSemantic proximity Read

Lipid-siRNA conjugate accesses a perivascular transport mechanism and achieves widespread and durable knockdown in the central nervous systemSemantic proximity Read

Self-amplifying mRNA expression is governed by mitochondrial machinery and ACSL4Semantic proximity Read

Controllable self-replicating RNA vaccine delivered intradermally elicits predominantly cellular immunitySemantic proximity Read

Functional mRNA delivery to hematopoietic stem and progenitor cells <i>in vivo</i>Semantic proximity Read

Engineering mRNA-LNP Medicines for the Ageing Brain: Opportunities and Challenges for Neurodegenerative diseasesSemantic proximity Read

Lipid nanoparticle-mediated delivery of mRNA into the mouse and human retina and other ocular tissuesSemantic proximity Read

Divergent Delivery and Expression Kinetics of Lipid and Polymeric Nanoparticles across mRNA ModalitiesSemantic proximity Read

Efficient genome editing in the non-human primate brain using programmable extracellular vesiclesSemantic proximity Read

A Lymphotropic Biomimetic Nanoplatform Reverses Sleep Deprivation-Induced Cognitive Deficits by Reprogramming Microglial PhenotypesSemantic proximity Read

Lipid nanoparticles for delivery of RNA therapeutics: Current status and the role of in vivo imagingSemantic proximity Read

Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteinsSemantic proximity Read