Abstract
Silencing gene expression by harnessing the RNA interference (RNAi) pathway with short interfering RNAs (siRNAs) has useful analytical and potentially therapeutic application. To augment silencing efficacy of siRNAs, chemical modification has been employed to improve stability, target specificity, and delivery to target tissues. siRNAs incorporating guanidinopropyl (GP) moieties have demonstrated enhanced target gene silencing in cell culture and in vivo models of hepatitis B virus replication. Here we describe the synthesis of GP-modified siRNAs and use of 5′ rapid amplification of cDNA ends (5′ RACE) to verify an RNAi-mediated mechanism of action of these novel chemically modified siRNAs.
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Acknowledgements
We gratefully acknowledge financial assistance from the Ernst & Ethel Eriksen Trust, National Research Foundation of South Africa (NRF), Medical Research Council, the Poliomyelitis Research Foundation (PRF) and from the German Research Foundation (DFG). We also thankfully acknowledge the helpful assistance of Corvin Steidinger, Christian Schuch, and Timo Weinrich in the synthesis of the GP-modified monomers.
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Buff, M.C.R., Bernhardt, S., Marimani, M.D., Ely, A., Engels, J.W., Arbuthnot, P. (2015). Use of Guanidinopropyl-Modified siRNAs to Silence Gene Expression. In: Sioud, M. (eds) RNA Interference. Methods in Molecular Biology, vol 1218. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-1538-5_13
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DOI: https://doi.org/10.1007/978-1-4939-1538-5_13
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