Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species

PURPOSE: Antisense oligonucleotides have been under investigation as potential therapeutics for many diseases, including inherited retinal diseases. Chemical modifications, such as chiral phosphorothioate (PS) backbone modification, are often used to improve stability and pharmacokinetic properties...

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Detalles Bibliográficos
Autores principales: Byrne, Michael, Vathipadiekal, Vinod, Apponi, Luciano, Iwamoto, Naoki, Kandasamy, Pachamuthu, Longo, Kenneth, Liu, Fangjun, Looby, Richard, Norwood, Lauren, Shah, Anee, Shelke, Juili Dilip, Shivalila, Chikdu, Yang, Hailin, Yin, Yuan, Guo, Lankai, Bowman, Keith, Vargeese, Chandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Association for Research in Vision and Ophthalmology 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7804567/
https://www.ncbi.nlm.nih.gov/pubmed/33510962
http://dx.doi.org/10.1167/tvst.10.1.23

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