Pharmacology & ToxicologyMolecular TherapyPeer-reviewed
Quantifying and Mitigating Motor Phenotypes Induced by Antisense Oligonucleotides in the Central Nervous System
M. P. Moazami, J. M. Rembetsy-Brown, F. Wang…·J. K. Watts
This study quantifies motor phenotypes caused by ASOs in the CNS and identifies modifications that reduce toxicity while maintaining efficacy.
DOI 10.1016/j.ymthe.2024.10.024