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Progress in MicroRNA Delivery

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PDF JCR-D-13-00424_2013-09-12.accepted.pdf (2MB) Main Article PDF
Title: Progress in MicroRNA Delivery
Author(s): Zhang, Yu; Wang, Zaijie; Gemeinhart, Richard A.
Subject(s): microRNA miRNA small RNA delivery
Abstract: MicroRNAs (miRNAs) are non-coding endogenous RNAs that direct post-transcriptional regulation of gene expression by several mechanisms. Activity is primarily through binding to the 3' untranslated regions (UTRs) of messenger RNAs (mRNA) resulting in degradation and translation repression. Unlike other small-RNAs, miRNAs do not require perfect base pairing, and thus, can regulate a network of broad, yet specific, genes. Although we have only just begun to gain insights into the full range of biologic functions of miRNA, their involvement in the onset and progression of disease has generated significant interest for therapeutic development. Mounting evidence suggests that miRNA-based therapies, either restoring or repressing miRNAs expression and activity, hold great promise. However, despite the early promise and exciting potential, critical hurdles often involving delivery of miRNA-targeting agents remain to be overcome before transition to clinical applications. Limitations that may be overcome by delivery include, but are not limited to, poor in vivo stability, inappropriate biodistribution, disruption and saturation of endogenous RNA machinery, and untoward side effects. Both viral vectors and nonviral delivery systems can be developed to circumvent these challenges. Viral vectors are efficient delivery agents but toxicity and immunogenicity limit their clinical usage. Herein, we review the recent advances in the mechanisms and strategies of nonviral miRNA delivery systems and provide a perspective on the future of miRNA-based therapeutics.
Issue Date: 2013-09
Publisher: Elsevier Inc.
Citation Info: Zhang Y, Wang Z, Gemeinhart RA. Progress in microRNA delivery. Journal of Controlled Release. 2013. doi:10.1016/j.jconrel.2013.09.015.
Type: Article
Description: NOTICE: This is the author’s version of a work that was accepted for publication in Journal of Controlled Release . Changes resulting from the publishing process, such as peer review, editing, corrections, structural formatting, and other quality control mechanisms may not be reflected in this document. Changes may have been made to this work since it was submitted for publication. A definitive version was subsequently published in Journal of Controlled Release , 2013 DOI:10.1016/j.jconrel.2013.09.015
ISSN: 1873-4995
Sponsor: This review and our original miRNA research was conducted in a facility constructed with support from Research Facilities Improvement Program Grant (RR15482) from the National Centre for Research Resources (NCRR) of the National Institutes of Health (NIH). Our original miRNA delivery research has been funded, in part, by the University of Illinois at Chicago Center for Clinical and Translational Science (CCTS) award also supported by the NCRR (TR000050, RAG).
Date Available in INDIGO: 2014-09-09

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