Effect of Extended Intracerebroventricular (ICV) Delivery on the Biodistribution of a Viral Vector in the CNS Of NHPs
1:30 pm - Thursday 3rd December 2026
- Achieving therapeutic levels in subcortical areas for the treatment of motor disorders is a daunting challenge in gene therapy
- This study compares bolus versus extended intracerebroventricular (ICV) administration schedules for delivering AAV vectors in cynomolgus macaques. Using a stereotactically implanted infusion system, animals received the same total vector dose either as two quick infusions or as eight smaller infusions over four hours
- The extended schedule led to greater gene expression in several brain regions, as well as peripheral tissues, suggesting that prolonged ICV infusion can enhance the distribution and expression of AAV-delivered genes in the CNS
- These findings support further exploration of extended infusion protocols to improve AAV gene therapy
