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Molecules 2018, 23(7), 1500; https://doi.org/10.3390/molecules23071500

MicroRNA-Regulated Gene Delivery Systems for Research and Therapeutic Purposes

1
Gallipoli Medical Research Institute, Greenslopes Private Hospital, 102 Newdegate Street, Brisbane, QLD 4120, Australia
2
Faculty of Medicine, University of Queensland, 288 Herston Road, Herston, Brisbane, QLD 4006, Australia
3
University of Queensland Diamantina Institute, Translational Research Institute, 37 Kent Street, Woolloongabba, QLD 4102, Australia
4
Layton Vision Foundation, Translational Research Institute, 37 Kent Street, Woolloongabba, QLD 4102, Australia
5
OcuGene, Translational Research Institute, 37 Kent Street, Woolloongabba, QLD 4102, Australia
*
Author to whom correspondence should be addressed.
Received: 30 May 2018 / Revised: 18 June 2018 / Accepted: 20 June 2018 / Published: 21 June 2018
(This article belongs to the Special Issue Gene Delivery)
Full-Text   |   PDF [781 KB, uploaded 21 June 2018]   |  

Abstract

Targeted gene delivery relies on the ability to limit the expression of a transgene within a defined cell/tissue population. MicroRNAs represent a class of highly powerful and effective regulators of gene expression that act by binding to a specific sequence present in the corresponding messenger RNA. Involved in almost every aspect of cellular function, many miRNAs have been discovered with expression patterns specific to developmental stage, lineage, cell-type, or disease stage. Exploiting the binding sites of these miRNAs allows for construction of targeted gene delivery platforms with a diverse range of applications. Here, we summarize studies that have utilized miRNA-regulated systems to achieve targeted gene delivery for both research and therapeutic purposes. Additionally, we identify criteria that are important for the effectiveness of a particular miRNA for such applications and we also discuss factors that have to be taken into consideration when designing miRNA-regulated expression cassettes. View Full-Text
Keywords: gene delivery; gene therapy; targeted transgene expression; microRNA; post-transcriptional targeting gene delivery; gene therapy; targeted transgene expression; microRNA; post-transcriptional targeting
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).
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Dhungel, B.; Ramlogan-Steel, C.A.; Steel, J.C. MicroRNA-Regulated Gene Delivery Systems for Research and Therapeutic Purposes. Molecules 2018, 23, 1500.

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