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 (Sn-: n = 5, 15, 20, 25, and 30 [number of spermine units]) as splice switching oligonucleotides (SSOs...

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Main Authors: Marc Nothisen (Author), Phanélie Perche-Létuvée (Author), Jean-Paul Behr (Author), Jean-Serge Remy (Author), Mitsuharu Kotera (Author)
Format: Book
Published: Elsevier, 2018-12-01T00:00:00Z.
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Call Number: A1234.567
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