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Gene Delivery and Gene Editing

Tech Session II: Gene Delivery and Gene Editing I

Unraveling endosomal escape: mechanistic insights into lipid nanoparticle-mediated RNA delivery

Tuesday, July 15, 2025
3:49 PM – 4:00 PM EDT
Introduction: Lipid nanoparticles (LNPs) represent the most promising platform for RNA delivery, yet their therapeutic efficacy is often limited by inefficient endosomal escape following cellular uptake [1-3]. Understanding the mechanisms that drive endosomal escape is critical to overcoming this barrier and optimizing LNP performance [4, 5]. Current models struggle to isolate and deconstruct the molecular events driving RNA release, leaving a critical gap in our understanding of this process.

Learning Objectives:

At the completion of this activity, participants will know
  • 1. Decode the molecular mechanisms underpinning LNP-mediated RNA delivery using GUV systems
  • 2. Identify design parameters and environmental conditions that optimize RNA release.
  • 3. Translate insights from GUV models to cellular systems to enhance therapeutic development