ArticleNAR molecular medicine2026
Systemic distribution of tricyclo-DNA antisense oligonucleotide following intratracheal instillation in the mouse.
Article in NAR molecular medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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Who cites it
1 citing paper in PubMed.
- Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application toMolecular therapy. Nucleic acids · 2026Article
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
This study evaluated intratracheal (IT) instillation as an alternative route for systemic delivery of therapeutic tricyclo-DNA antisense oligonucleotides (tcDNA-ASOs), using the Duchenne muscular dystrophy (DMD)-targeting compound SQY51 as a model. IT administration was compared with intravenous (IV) injection by assessing pharmacokinetics, biodistribution, tissue localization, and immunological effects in mice. Fluorescent hybridization assays demonstrated that IT instillation delivered SQY51 into the bloodstream, confirming systemic absorption via the pulmonary route. Compared with IV injection, IT administration produced lower peak plasma concentrations, slower clearance, and prolonged detection. Tissue distribution analysis showed higher lung accumulation but reduced levels in the kidney, heart, and diaphragm, indicating route-dependent targeting. Histological and molecular analyses showed preserved lung architecture and no significant inflammation after IT delivery. Unlike carbon black, a known inflammatory agent, SQY51 did not induce pro-inflammatory cytokine expression
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Registered trials
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