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Achieving HIV-1 Control through RNA-Directed Gene Regulation

1
Kirby Institute, The University of New South Wales, Sydney, NSW 2052, Australia
2
ARC Centre of Excellence in Convergent Bio-Nano Science and Technology, The University of Melbourne, Parkville, Victoria 3010, Australia
3
Department of Chemical and Biomolecular Engineering, The University of Melbourne, Parkville, Victoria 3010, Australia
4
Calimmune Inc., Darlinghurst, NSW 2011, Australia
*
Author to whom correspondence should be addressed.
Academic Editor: Wenyi Gu
Genes 2016, 7(12), 119; https://doi.org/10.3390/genes7120119
Received: 13 October 2016 / Revised: 28 November 2016 / Accepted: 29 November 2016 / Published: 7 December 2016
(This article belongs to the Special Issue RNA Interference 2016)
HIV-1 infection has been transformed by combined anti-retroviral therapy (ART), changing a universally fatal infection into a controllable infection. However, major obstacles for an HIV-1 cure exist. The HIV latent reservoir, which exists in resting CD4+ T cells, is not impacted by ART, and can reactivate when ART is interrupted or ceased. Additionally, multi-drug resistance can arise. One alternate approach to conventional HIV-1 drug treatment that is being explored involves gene therapies utilizing RNA-directed gene regulation. Commonly known as RNA interference (RNAi), short interfering RNA (siRNA) induce gene silencing in conserved biological pathways, which require a high degree of sequence specificity. This review will provide an overview of the silencing pathways, the current RNAi technologies being developed for HIV-1 gene therapy, current clinical trials, and the challenges faced in progressing these treatments into clinical trials. View Full-Text
Keywords: HIV-1; RNAi; transcriptional gene silencing; gene therapy; clinical trials HIV-1; RNAi; transcriptional gene silencing; gene therapy; clinical trials
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Klemm, V.; Mitchell, J.; Cortez-Jugo, C.; Cavalieri, F.; Symonds, G.; Caruso, F.; Kelleher, A.D.; Ahlenstiel, C. Achieving HIV-1 Control through RNA-Directed Gene Regulation. Genes 2016, 7, 119.

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