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Viruses 2018, 10(1), 8; doi:10.3390/v10010008

RNA Interference Therapies for an HIV-1 Functional Cure

Lady Davis Institute for Medical Research, Montreal, QC H3T 1E2, Canada
Department of Microbiology and Immunology, McGill University, Montreal, QC H3A0G4, Canada
Department of Medicine, Division of Experimental Medicine, McGill University, Montreal, QC H3A0G4, Canada
Author to whom correspondence should be addressed.
Received: 30 November 2017 / Revised: 20 December 2017 / Accepted: 22 December 2017 / Published: 27 December 2017
(This article belongs to the Special Issue Homage to Mark Wainberg)
View Full-Text   |   Download PDF [2317 KB, uploaded 27 December 2017]   |  


HIV-1 drug therapies can prevent disease progression but cannot eliminate HIV-1 viruses from an infected individual. While there is hope that elimination of HIV-1 can be achieved, several approaches to reach a functional cure (control of HIV-1 replication in the absence of drug therapy) are also under investigation. One of these approaches is the transplant of HIV-1 resistant cells expressing anti-HIV-1 RNAs, proteins or peptides. Small RNAs that use RNA interference pathways to target HIV-1 replication have emerged as competitive candidates for cell transplant therapy and have been included in all gene combinations that have so far entered clinical trials. Here, we review RNA interference pathways in mammalian cells and the design of therapeutic small RNAs that use these pathways to target pathogenic RNA sequences. Studies that have been performed to identify anti-HIV-1 RNA interference therapeutics are also reviewed and perspectives on their use in combination gene therapy to functionally cure HIV-1 infection are provided. View Full-Text
Keywords: HIV-1; functional cure; cell transplant; RNA interference; small/short interfering RNA; small/short hairpin RNA; micro RNA HIV-1; functional cure; cell transplant; RNA interference; small/short interfering RNA; small/short hairpin RNA; micro RNA

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Scarborough, R.J.; Gatignol, A. RNA Interference Therapies for an HIV-1 Functional Cure. Viruses 2018, 10, 8.

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