Therapeutic Genome Editing of Gliomas via Focused Ultrasound-Mediated Ribonucleoprotein Delivery
Friday, July 18, 2025
11:30 AM - 11:50 AM EDT
Location: Terrace Ballroom 3/4
Introduction: Efflux pumps (e.g. P-glycoprotein (P-gp)) are hallmarks of the blood-brain barrier that significantly hinder chemotherapy delivery to glioblastoma (GBM) (1). Pharmacological P-gp inhibitors have limited utility because blocking P-gp in healthy tissues yields heightened chemotherapy toxicity. A non-invasive and spatially-targeted strategy to block P-gp would have substantial clinical impact. Here, we developed a focused ultrasound (FUS)-targeted delivery approach for ribonucleoprotein (RNP) CRISPR/Cas9-based genomic deletion of P-gp in GBM that augments the efficacy of chemotherapy.
Learning Objectives:
At the completion of this activity, participants will know
Understand the role of P-glycoprotein in limiting chemotherapy delivery.
Evaluate the use of lung SORT LNPs for minimizing off-target P-gp editing.
Assess the potential of FUS-mediated delivery of RNPs for overcoming chemotherapy resistance in GBM.
Victoria Breza, BS – PhD Candidate, University of Virginia; Catherine Gorick, PhD – Senior Research Scientist, University of Virginia; Richard Price, PhD – Principal Investigator, University of Virginia; Ji Song, PhD – Lab Manager, University of Virginia
