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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...

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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
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