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2′-O-(N-(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity
2′-O-(N-(Aminoethyl)carbamoyl)methyl (2′-O-AECM)-modified oligonucleotides (ONs) and their mixmers with 2′-O-methyl oligonucleotides (2′-OMe ONs) with phosphodiester linkers as well as with partial and full phosphorothioate (PS) inclusion were synthesized and functionally evaluated as splice-switchi...
Autores principales: | Honcharenko, Dmytro, Rocha, Cristina S.J., Lundin, Karin E., Maity, Jyotirmoy, Milton, Stefan, Tedebark, Ulf, Murtola, Merita, Honcharenko, Malgorzata, Slaitas, Andis, Smith, C.I. Edvard, Zain, Rula, Strömberg, Roger |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Mary Ann Liebert, Inc., publishers
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9221157/ https://www.ncbi.nlm.nih.gov/pubmed/35238623 http://dx.doi.org/10.1089/nat.2021.0086 |
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