The nucleobase guanine at the 3’-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity

Valencia, T., Yen, L. Y., Berman, C., Vincent, T., Davis, S., Varrone, F., Huang, J., Mastroianni, J., Carlson, M., Owen, T., Kamel, A., Drygin, D., Kinberger, G. A., Gangwar, S. P., Yelshanskaya, M. V., Ridley, J., Kirby, R., Alvarez, J., Lakhia, R., ... Lee, E. C. (2026). The nucleobase guanine at the 3’-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity. Nature Communications. DOI: https://doi.org/10.1038/s41467-025-65799-5 [Article] [DOI]