Pharmacology & ToxicologybioRxiv
Intrathecal sc-AAV9-CB-GFP: Systemic Distribution Predominates Following Single-Dose Administration in Cynomolgus Macaques
E. K. Meseck, G. Guibinga, S. Wang…·K. Mansfield
This study evaluates the biodistribution of scAAV9-CB-GFP in cynomolgus macaques after intrathecal administration, showing systemic spread and limited brain neuronal transduction.
DOI 10.1177/01926233221101309