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Achieving HIV-1 Control through RNA-Directed Gene Regulation

Kirby Institute, The University of New South Wales, Sydney, NSW 2052, Australia
ARC Centre of Excellence in Convergent Bio-Nano Science and Technology, The University of Melbourne, Parkville, Victoria 3010, Australia
Department of Chemical and Biomolecular Engineering, The University of Melbourne, Parkville, Victoria 3010, Australia
Calimmune Inc., Darlinghurst, NSW 2011, Australia
Author to whom correspondence should be addressed.
Academic Editor: Wenyi Gu
Genes 2016, 7(12), 119;
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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MDPI and ACS Style

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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