Control of backbone chemistry and chirality boost oligonucleotide splice switching activity

Although recent regulatory approval of splice-switching oligonucleotides (SSOs) for the treatment of neuromuscular disease such as Duchenne muscular dystrophy has been an advance for the splice-switching field, current SSO chemistries have shown limited clinical benefit due to poor pharmacology. To...

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Detalles Bibliográficos
Autores principales: Kandasamy, Pachamuthu, McClorey, Graham, Shimizu, Mamoru, Kothari, Nayantara, Alam, Rowshon, Iwamoto, Naoki, Kumarasamy, Jayakanthan, Bommineni, Gopal R, Bezigian, Adam, Chivatakarn, Onanong, Butler, David C D, Byrne, Michael, Chwalenia, Katarzyna, Davies, Kay E, Desai, Jigar, Shelke, Juili Dilip, Durbin, Ann F, Ellerington, Ruth, Edwards, Ben, Godfrey, Jack, Hoss, Andrew, Liu, Fangjun, Longo, Kenneth, Lu, Genliang, Marappan, Subramanian, Oieni, Jacopo, Paik, Ik-Hyeon, Estabrook, Erin Purcell, Shivalila, Chikdu, Tischbein, Maeve, Kawamoto, Tomomi, Rinaldi, Carlo, Rajão-Saraiva, Joana, Tripathi, Snehlata, Yang, Hailin, Yin, Yuan, Zhao, Xiansi, Zhou, Cong, Zhang, Jason, Apponi, Luciano, Wood, Matthew J A, Vargeese, Chandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Chemical Biology and Nucleic Acid Chemistry
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9178015/
https://www.ncbi.nlm.nih.gov/pubmed/35061895
http://dx.doi.org/10.1093/nar/gkac018

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