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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...
Autores principales: | Pedersen, Lykke, Hagedorn, Peter, Vikeså, Jonas, Karlsen, Signe Tang, Særmark, Pia, Koch, Troels, Lindow, Morten |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2019
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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 |
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