Abstract
Small interfering RNA (SiRNA) delivery remains a major challenge in RNAi-based therapy. Dendrimers are emerging as appealing nonviral vectors for SiRNA delivery thanks to their well-defined architecture and their unique cooperativity and multivalency confined within a nanostructure. We have recently demonstrated that structurally flexible poly(amidoamine) (PAMAM) dendrimers are safe and effective nanovectors for SiRNA delivery in various disease models in vitro and in vivo. The present chapter showcases these dendrimers can package different SiRNA molecules into stable and nanosized particles, which protect SiRNA from degradation and promote cellular uptake of SiRNA, resulting in potent gene silencing at both mRNA and protein level in the prostate cancer cell model. Our results demonstrate this set of flexible PAMAM dendrimers are indeed safe and effective nonviral vectors for SiRNA delivery and hold great promise for further applications in functional genomics and RNAi-based therapies.
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Acknowledgments
This work was supported by the international ERA-Net EURONANOMED European Research project DENANORNA, PACA Canceropôle, INCa, Aix-Marseille Université, CNRS, INSERM, China Scholarship Council (XL), Association pour la Recherche sur les Tumeurs de la Prostate (XL), Association Française contre les Myopathies (XL), and under the auspice of European COST Action TD0802 “Dendrimers in Biomedical Applications.”
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Liu, X., Peng, L. (2016). Dendrimer Nanovectors for SiRNA Delivery. In: Shum, K., Rossi, J. (eds) SiRNA Delivery Methods. Methods in Molecular Biology, vol 1364. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-3112-5_11
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DOI: https://doi.org/10.1007/978-1-4939-3112-5_11
Publisher Name: Humana Press, New York, NY
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