Oligonucleotides, Antisense

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Thakur, P., Lackinger, M., Diamantopoulou, A., Rao, S., Chen, Y., Khalizova, K., Ferng, A., Mazur, C., Kordasiewicz, H., Shprintzen, R. J., Markx, S., Xu, B., & Gogos, J. A. (2025). An antisense oligonucleotide-based strategy to ameliorate cognitive dysfunction in the 22q11.2 Deletion Syndrome. ELife, 13. CLOCKSS. https://doi.org/10.7554/elife.103328
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Miller, T. M., Cudkowicz, M. E., Genge, A., Shaw, P. J., Sobue, G., Bucelli, R. C., Chiò, A., Van Damme, P., Ludolph, A. C., Glass, J. D., Andrews, J. A., Babu, S., Benatar, M., McDermott, C. J., Cochrane, T., Chary, S., Chew, S., Zhu, H., Wu, F., … Fradette, S. (2022). Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. New England Journal of Medicine, 387(12), 1099–1110. https://doi.org/10.1056/nejmoa2204705
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