Reversable Acute Sedation Response of Phosphorothioate Antisense Oligonucleotides Following Local Delivery to the Central Nervous System
Jacqueline G. O’Rourke, Gemma Bachmann, Curt Mazur, Keming Zhou, Oleksandr Platoshyn, Mariana Bravo Hernandez, Stéphanie Klein, Jonathon Nguyen, Sebastien A. Burel, Christine M. Hoffmaster, Tom A. Zanardi, Paymaan Jafar‐Nejad, Martin Maršala, Scott P. Henry, Eric E. Swayze, , Holly B. Kordasiewicz
This study characterizes an acute sedation response caused by phosphorothioate ASOs after CNS delivery, showing it is sequence-, dose-, and chemistry-dependent and can be mitigated.
bioRxiv · Feb 13, 2025 · PreprintNeuroscience