Dorsal Root Ganglia Pathology in AAV Preclinical Studies: Meta-Analysis & Mitigation Strategy

Time: 8:05 am
day: Day Two


• The administration of adeno-associated virus (AAV) vectors to nonhuman primates (NHP) can lead to dorsal root ganglion (DRG) pathology
• A meta-analysis including 256 NHP shows that 83% of animals that were administered AAV through the cerebrospinal fluid (CSF), and 32% of those that received an intravenous (IV) injection developed some DRG pathology and secondary axonopathy
• We developed an efficacious mitigation strategy using DRG-specific microRNA targets