Abstract
RNA interference (RNAi) mediated by small interfering RNA (SiRNA) has recently emerged as a potent machinery in regulating gene expression at the post-translation step. Despite its efficiency and specificity, the biggest hurdle against its wide application is the safe and effective delivery of the SiRNA cargo into target cells. Here, we describe the highly effective SiRNA delivery mediated by the cationic helical polypeptides and polypeptide-based nanosystems both in vitro and in vivo via oral administration.
This is a preview of subscription content, log in via an institution to check access.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Davis ME, Zuckerman JE, Choi CHJ, Seligson D, Tolcher A, Alabi CA, Yen Y, Heidel JD, Ribas A (2010) Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature 464:1067–1070
Lee H, Lytton-Jean AKR, Chen Y, Love KT, Park AI, Karagiannis ED, Sehgal A, Querbes W, Zurenko CS, Jayaraman M, Peng CG, Charisse K, Borodovsky A, Manoharan M, Donahoe JS, Truelove J, Nahrendorf M, Langer R, Anderson DG (2012) Molecularly self-assembled nucleic acid nanoparticles for targeted in vivo siRNA delivery. Nat Nanotechnol 7:389–393
Woodrow KA, Cu Y, Booth CJ, Saucier-Sawyer JK, Wood MJ, Saltzman WM (2009) Intravaginal gene silencing using biodegradable polymer nanoparticles densely loaded with small-interfering RNA. Nat Mater 8:526–533
Dassie JP, Liu XY, Thomas GS, Whitaker RM, Thiel KW, Stockdale KR, Meyerholz DK, McCaffrey AP, McNamara JO, Giangrande PH (2009) Systemic administration of optimized aptamer-siRNA chimeras promotes regression of PSMA-expressing tumors. Nat Biotechnol 27:839–849
Gabrielson NP, Lu H, Yin LC, Li D, Wang F, Cheng JJ (2012) Reactive and bioactive cationic α-helical polypeptide template for nonviral gene delivery. Angew Chem Int Ed Engl 51:1143–1147
Gabrielson NP, Lu H, Yin LC, Kim KH, Cheng JJ (2012) A cell-penetrating helical polymer for siRNA delivery to mammalian cells. Mol Ther 20:1599–1609
Yin LC, Song ZY, Qu QH, Kim KH, Zheng N, Yao C, Chaudhury I, Tang HY, Gabrielson NP, Uckun FM, Cheng JJ (2013) Supramolecular self-assembled nanoparticles mediate oral delivery of therapeutic TNF-alpha siRNA against systemic inflammation. Angew Chem Int Ed Engl 52:5757–5761
Filleur S, Courtin A, Ait-Si-Ali S, Guglielmi J, Merle C, Harel-Bellan A, Clezardin P, Cabon F (2003) SiRNA-mediated inhibition of vascular endothelial growth factor severely limits tumor resistance to antiangiogenic thrombospondin-1 and slows tumor vascularization and growth. Cancer Res 63:3919–3922
McCaffrey AP, Meuse L, Pham TTT, Conklin DS, Hannon GJ, Kay MA (2002) Gene expression—RNA interference in adult mice. Nature 418:38–39
Soutschek J, Akinc A, Bramlage B, Charisse K, Constien R, Donoghue M, Elbashir S, Geick A, Hadwiger P, Harborth J, John M, Kesavan V, Lavine G, Pandey RK, Racie T, Rajeev KG, Rohl I, Toudjarska I, Wang G, Wuschko S, Bumcrot D, Koteliansky V, Limmer S, Manoharan M, Vornlocher HP (2004) Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432:173–178
Author information
Authors and Affiliations
Corresponding authors
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2016 Springer Science+Business Media New York
About this protocol
Cite this protocol
Yin, L., Zheng, N., Cheng, J. (2016). Highly Efficient SiRNA Delivery Mediated by Cationic Helical Polypeptides and Polypeptide-Based Nanosystems. 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_4
Download citation
DOI: https://doi.org/10.1007/978-1-4939-3112-5_4
Publisher Name: Humana Press, New York, NY
Print ISBN: 978-1-4939-3111-8
Online ISBN: 978-1-4939-3112-5
eBook Packages: Springer Protocols