Cationic Oligospermine-Oligonucleotide Conjugates Provide Carrier-free Splice Switching in Monolayer Cells and Spheroids

We report the evaluation of 18-mer 2′-O-methyl-modified ribose oligonucleotides with a full-length phosphorothioate backbone chemically conjugated at the 5′ end to the oligospermine units (S(n)-: n = 5, 15, 20, 25, and 30 [number of spermine units]) as splice switching oligonucleotides (SSOs). These...

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Detalles Bibliográficos
Autores principales: Nothisen, Marc, Perche-Létuvée, Phanélie, Behr, Jean-Paul, Remy, Jean-Serge, Kotera, Mitsuharu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6205332/
https://www.ncbi.nlm.nih.gov/pubmed/30388622
http://dx.doi.org/10.1016/j.omtn.2018.09.027

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6205332/
https://www.ncbi.nlm.nih.gov/pubmed/30388622
http://dx.doi.org/10.1016/j.omtn.2018.09.027

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