Impact of guanidine-containing backbone linkages on stereopure antisense oligonucleotides in the CNS

Attaining sufficient tissue exposure at the site of action to achieve the desired pharmacodynamic effect on a target is an important determinant for any drug discovery program, and this can be particularly challenging for oligonucleotides in deep tissues of the CNS. Herein, we report the synthesis a...

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Detalles Bibliográficos
Autores principales: Kandasamy, Pachamuthu, Liu, Yuanjing, Aduda, Vincent, Akare, Sandheep, Alam, Rowshon, Andreucci, Amy, Boulay, David, Bowman, Keith, Byrne, Michael, Cannon, Megan, Chivatakarn, Onanong, Shelke, Juili Dilip, Iwamoto, Naoki, Kawamoto, Tomomi, Kumarasamy, Jayakanthan, Lamore, Sarah, Lemaitre, Muriel, Lin, Xuena, Longo, Kenneth, Looby, Richard, Marappan, Subramanian, Metterville, Jake, Mohapatra, Susovan, Newman, Bridget, Paik, Ik-Hyeon, Patil, Saurabh, Purcell-Estabrook, Erin, Shimizu, Mamoru, Shum, Pochi, Standley, Stephany, Taborn, Kris, Tripathi, Snehlata, Yang, Hailin, Yin, Yuan, Zhao, Xiansi, Dale, Elena, Vargeese, Chandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
NAR Breakthrough Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9177980/
https://www.ncbi.nlm.nih.gov/pubmed/35106589
http://dx.doi.org/10.1093/nar/gkac037

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