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FAP-Synergistic Organ-Targeted mRNA-LNP for Overcoming Delivery Barriers in Hepatic and Pulmonary Fibrosis - PubMed

4 hours ago
  • #lipid nanoparticles
  • #fibrosis therapy
  • #mRNA delivery
  • Developed FOCUS (Fibrosis Organ and Cell Unified-Targeting System) for mRNA delivery using LNPs (lipid nanoparticles).
  • Combines ionizable lipid library with FAP-targeting ligands for enhanced mRNA delivery to fibrotic tissues.
  • Achieves 3-fold and 12-fold greater mRNA expression in fibrotic liver and lung, respectively.
  • Demonstrates 2-5-fold higher fibroblast distribution compared to organ- or ligand-only targeting.
  • Proof of concept: mPTX-2-loaded FOCUS LNPs reduce collagen deposition by 60-80% in murine models.
  • Avoids systemic side effects like impaired wound healing.
  • Mechanism: Suppresses monocyte-to-fibrocyte differentiation, M2 macrophage polarization, and fibroblast activation.
  • Establishes a precision mRNA delivery platform for safer and more effective fibrotic disease therapy.