Abstract
In this chapter, an overview of chemically modified antisense oligonucleotides (AON) for Duchenne muscular dystrophy (DMD) treatment is described. In particular, the promising exon-skipping properties of 2′-O-modified phosphorothioate oligoribonucleotides incorporating 2′-O-[2-(N-methycarbamoyl)ethyl]ribonucleosides that could be readily synthesized by new regioselective oxa-Michael reaction are reviewed in detail.
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Masaki, Y., Yamada, T., Saneyoshi, H., Ohkubo, A., Seio, K., Sekine, M. (2014). Synthesis and Exon-Skipping Activity of Chemically Modified RNAs. In: Erdmann, V., Markiewicz, W., Barciszewski, J. (eds) Chemical Biology of Nucleic Acids. RNA Technologies. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-54452-1_27
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DOI: https://doi.org/10.1007/978-3-642-54452-1_27
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