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Targeting Repeated Regions Unique to a Gene Is an Effective Strategy for Discovering Potent and Efficacious Antisense Oligonucleotides

Well-validated strategies for discovering potent and efficacious antisense oligonucleotides are central to realize the full therapeutic potential of RNA therapy. In this study, we focus on RNA targets where the same sequence of 16–20 nt is found in several regions across the RNA, and not in any othe...

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Detalles Bibliográficos
Autores principales: Pedersen, Lykke, Hagedorn, Peter, Vikeså, Jonas, Karlsen, Signe Tang, Særmark, Pia, Koch, Troels, Lindow, Morten
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6921215/
https://www.ncbi.nlm.nih.gov/pubmed/31846799
http://dx.doi.org/10.1016/j.omtn.2019.10.040