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Nature-inspired IL-1 targeted therapy to treat chronic inflammatory diseases

GSE305182 Mus musculus Expression profiling by high throughput sequencing 6 samples Submitted 2025/10/31 Platform GPL28457
Summary
The interleukin-1 (IL-1) pathway drives inflammation in rheumatoid arthritis (RA) and other autoinflammatory diseases. We developed a recombinant adeno-associated virus (rAAV) gene therapy that delivers a secreted human IL-1 receptor antagonist (sIL-1Ra) under an inflammation-inducible promoter. This vector responds to pro-inflammatory cytokines and BMPs enriched in inflamed joints, enabling on-demand sIL-1Ra production. In mouse models of RA and IL-1Ra deficiency, inflammation-inducible sIL-1Ra reduced IL-1 signaling, immune cell activation, inflammatory gene expression, joint swelling, and bone destruction. A single systemic dose provided long-lasting therapeutic benefit, supporting this approach as a targeted treatment for chronic inflammatory diseases.
Published in
Nature-inspired IL-1 targeted therapy to treat chronic inflammatory diseases
Yang YS, Kim MJ, Chaugule S et al. · Molecular therapy : the journal of the American Society of Gene Therapy 2025 · PMID 40931517 · doi:10.1016/j.ymthe.2025.09.008
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Also filed as BioProject PRJNA1304548 and SRA study SRP608208. Searching any of these in the dataset finder brings you back here.

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