Tech Session VII: Gene Delivery and Gene Editing IV
In vivo nucleic acid targeted delivery in bone-marrow via antibody conjugated lipid nanoparticles
Friday, July 18, 2025
9:16 AM – 9:27 AM EDT
Introduction: Delivering therapeutic payloads to extrahepatic tissues via lipid nanoparticles (LNPs) is often hindered by their tendency to accumulate in liver. Traditional gene therapy for hematopoietic disorders involves isolating bone marrow (BM) cells from patients, ex vivo gene editing and reinfusing edited BM cells into patients. This is further complicated by systemic toxicities associated with BM conditioning. To overcome these challenges, we developed antibody-conjugated targeted LNPs (tLNPs) to deliver mRNAs directly into BM, facilitating in vivo gene editing for enhanced therapeutic outcomes.
Learning Objectives:
At the completion of this activity, participants will know
Identify challenges of LNP-mediated gene delivery to extrahepatic tissues and potential solutions.
Learn that ligand targeted LNPs may treat genetic disorders in vivo, overcoming present challenges.
Conduct enhanced delivery of functional mRNA into bone-marrow via tLNPs, and de-target liver.
