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Viruses 2015, 7(7), 3910-3936; doi:10.3390/v7072804

Bone Marrow Gene Therapy for HIV/AIDS

Laboratory of Experimental Virology, Department of Medical Microbiology, Center for Infection and Immunity Amsterdam (CINIMA), Academic Medical Center, University of Amsterdam, Amsterdam 1105 AZ, The Netherlands
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Author to whom correspondence should be addressed.
Academic Editor: Eric O. Freed
Received: 11 May 2015 / Revised: 9 July 2015 / Accepted: 13 July 2015 / Published: 17 July 2015
(This article belongs to the Special Issue Gene Technology and Resistance to Viruses - Reviews)
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Abstract

Bone marrow gene therapy remains an attractive option for treating chronic immunological diseases, including acquired immunodeficiency syndrome (AIDS) caused by human immunodeficiency virus (HIV). This technology combines the differentiation and expansion capacity of hematopoietic stem cells (HSCs) with long-term expression of therapeutic transgenes using integrating vectors. In this review we summarize the potential of bone marrow gene therapy for the treatment of HIV/AIDS. A broad range of antiviral strategies are discussed, with a particular focus on RNA-based therapies. The idea is to develop a durable gene therapy that lasts the life span of the infected individual, thus contrasting with daily drug regimens to suppress the virus. Different approaches have been proposed to target either the virus or cellular genes encoding co-factors that support virus replication. Some of these therapies have been tested in clinical trials, providing proof of principle that gene therapy is a safe option for treating HIV/AIDS. In this review several topics are discussed, ranging from the selection of the antiviral molecule and the viral target to the optimal vector system for gene delivery and the setup of appropriate preclinical test systems. The molecular mechanisms used to formulate a cure for HIV infection are described, including the latest antiviral strategies and their therapeutic applications. Finally, a potent combination of anti-HIV genes based on our own research program is described. View Full-Text
Keywords: bone marrow; hematopoietic stem cell (HSC); virus; antiviral; gene therapy; lentiviral vector; HIV-1; RNAi bone marrow; hematopoietic stem cell (HSC); virus; antiviral; gene therapy; lentiviral vector; HIV-1; RNAi
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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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Herrera-Carrillo, E.; Berkhout, B. Bone Marrow Gene Therapy for HIV/AIDS. Viruses 2015, 7, 3910-3936.

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