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Nanoparticle-Based Delivery of RNAi Therapeutics: Progress and Challenges

by Jiehua Zhou 1,†, Ka-To Shum 1,†, John C. Burnett 1 and John J. Rossi 1,2,*
1
Department of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, City of Hope, 1500 East Duarte Rd, Duarte, CA 91010, USA
2
Irell and Manella Graduate School of Biological Sciences, Beckman Research Institute of City of Hope, City of Hope, 1500 East Duarte Rd, Duarte, CA 91010, USA
*
Author to whom correspondence should be addressed.
These two authors contributed equally to this work.
Pharmaceuticals 2013, 6(1), 85-107; https://doi.org/10.3390/ph6010085
Received: 21 December 2012 / Revised: 8 January 2013 / Accepted: 14 January 2013 / Published: 16 January 2013
(This article belongs to the Special Issue RNAi-Based Therapeutics)
RNA interference (RNAi) is an evolutionarily conserved, endogenous process for post-transcriptional regulation of gene expression. Although RNAi therapeutics have recently progressed through the pipeline toward clinical trials, the application of these as ideal, clinical therapeutics requires the development of safe and effective delivery systems. Inspired by the immense progress with nanotechnology in drug delivery, efforts have been dedicated to the development of nanoparticle-based RNAi delivery systems. For example, a precisely engineered, multifunctional nanocarrier with combined passive and active targeting capabilities may address the delivery challenges for the widespread use of RNAi as a therapy. Therefore, in this review, we introduce the major hurdles in achieving efficient RNAi delivery and discuss the current advances in applying nanotechnology-based delivery systems to overcome the delivery hurdles of RNAi therapeutics. In particular, some representative examples of nanoparticle-based delivery formulations for targeted RNAi therapeutics are highlighted. View Full-Text
Keywords: small interfering (si)RNA; non-viral vector; multifunctional nanoparticle; targeting delivery; passive and active targeting small interfering (si)RNA; non-viral vector; multifunctional nanoparticle; targeting delivery; passive and active targeting
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Zhou, J.; Shum, K.-T.; Burnett, J.C.; Rossi, J.J. Nanoparticle-Based Delivery of RNAi Therapeutics: Progress and Challenges. Pharmaceuticals 2013, 6, 85-107.

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