Technical Note: Focused ultrasound-mediated blood-brain barrier opening for delivery of LNP-packaged modRNA therapy in a mouse model of Niemann-Pick Disease Type C
Nick Todd, Bridget Funk, Paige Nowlin, Christina Hung, Olaf A. Bodamer
This study evaluates FUS-BBBO for delivering LNP-packaged modRNA to the cerebellum in NPC mice, finding BBB opening but no detectable protein expression or therapeutic benefit.
bioRxiv · Jul 1, 2026 · PreprintBioengineering