Review

Gene Therapy (2007) 14, 1057–1064; doi:10.1038/sj.gt.3302977

Progress and prospects: RNA-based therapies for treatment of HIV infection

L Scherer1, J J Rossi1 and M S Weinberg2

  1. 1Division of Molecular Biology, City of Hope Beckman Research Institute, Duarte, CA, USA
  2. 2Department of Molecular Medicine and Hematology, University of the Witwatersrand Medical School, Wits, South Africa

Correspondence: Dr JJ Rossi, Division of Molecular Biology, Beckman Research Institute, Division of Molecular Biology Beckman Research Institute of the City of Hope, 1450 E. Duarte Rd, Duarte, CA, 91010, USA. E-mail: jrossi@bricoh.edu or jrossi@coh.org

Received 9 April 2007; Revised 12 May 2007; Accepted 12 May 2007.

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Abstract

The current treatment regimen for HIV-infected individuals combines two or more drugs targeting different viral proteins such as RT and gag. Resistance to conventional drugs can develop quickly, and typically persists. The prospect of longer, continuous antiretroviral therapy brings with it the need for new antiretroviral drugs and approaches. In this context, gene therapies have the potential to prolong life and quality of life as an additional therapeutic class and may serve as an adjuvant to traditional treatments. This review focuses on RNA-based hematopoietic cell gene therapy for treatment of HIV infection. Recent advances in our understanding of RNA interference (RNAi) make this an especially attractive candidate for anti-HIV gene therapy although ribozyme and RNA decoy/aptamer approaches can be combined with RNAi to make a combinatorial therapy akin to highly active anti-retroviral therapy.

Keywords:

therapy, RNAi, siRNA, ribozyme, aptamer, HIV-1

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