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July 30, 2026The Journal of Immunology

Therapeutic Evaluation of a C3-Targeting Small Nucleic Acid Drug in a Spontaneous Humanized C3 Glomerulopathy Mouse Model 2256845

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Authors

JLJuan LiangKZKaiyuan Zi

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Overview

Randomized trial evaluates drug efficacy in C3 glomerulopathy mouse model, suggesting new treatment pathways.

Key Points

  • This research aims to evaluate the effectiveness of a C3-targeting small nucleic acid drug in a mouse model of C3 glomerulopathy.
  • Established a humanized C3 glomerulopathy model (B6-hC3 mC3 KO) that develops progressive glomerulopathy.
  • Administered small nucleic acid drugs subcutaneously to evaluate their therapeutic effects.
  • Monitored serum C3 levels, plasma C3a levels, and renal histology after 6 weeks of treatment.
  • Subcutaneous administration of the drug reduced serum C3 and plasma C3a levels.
  • Histological analysis revealed markedly reduced deposition of hC3 and diminished inflammatory cell infiltration.
  • The model demonstrated key features of C3 glomerulopathy, affirming its utility for testing complement-targeted therapies.

Cite This Study

Liang et al. (2026) studied this question.

synapsesocial.com/papers/6a6af4e960e2b924d3ea06d6https://doi.org/10.1093/jimmun/vkag141.364
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