296. Longitudinal Surveillance of Non Human Primate Recipients of Intrauterine Gene Transfer for Coagulation Factor Deficiencies

Abstract

site and comparatively less leakage to peripheral organs compared with bolus injection. Preliminary experiments delivering rAAV9-ABCD1 at 0.5X10 11 GC by intracerebroventricular approach suggest behavioral improvement in the Abcd1-/-mouse despite localized expression in brain. We therefore anticipate even better performance at this dose using the outlined intrathecal pump delivery. We conclude that rAAV9-mediated ABCD1 gene transfer via intrathecal osmotic pump leads to more uniform and widespread gene delivery to CNS with reduced leakage into the systemic circulation compared with intrathecal bolus injection.