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Genetic Modification of Hematopoietic Stem Cells as a Therapy for HIV/AIDS
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Gene Therapy Strategies for HIV/AIDS: Preclinical Modeling in Humanized Mice

Department of Microbiology, Immunology and Pathology, Colorado State University, 1619 Campus delivery, Fort Collins, CO 80523, USA
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Viruses 2013, 5(12), 3119-3141; https://doi.org/10.3390/v5123119
Received: 6 September 2013 / Revised: 4 November 2013 / Accepted: 3 December 2013 / Published: 12 December 2013
(This article belongs to the Special Issue Gene Therapy for Retroviral Infections)
In the absence of an effective vaccine and lack of a complete cure, gene therapy approaches to control HIV infection offer feasible alternatives. Due to the chronic nature of infection, a wide window of opportunity exists to gene modify the HIV susceptible cells that continuously arise from the bone marrow source. To evaluate promising gene therapy approaches that employ various anti-HIV therapeutic molecules, an ideal animal model is necessary to generate important efficacy and preclinical data. In this regard, the humanized mouse models that harbor human hematopoietic cells susceptible to HIV infection provide a suitable in vivo system. This review summarizes the currently used humanized mouse models and different anti-HIV molecules utilized for conferring HIV resistance. Humanized mouse models are compared for their utility in this context and provide perspectives for new directions. View Full-Text
Keywords: gene therapy for HIV/AIDS; humanized mice for HIV gene therapy; stem cell-based gene therapy; anti-HIV RNA-based therapies; new generation humanized mice gene therapy for HIV/AIDS; humanized mice for HIV gene therapy; stem cell-based gene therapy; anti-HIV RNA-based therapies; new generation humanized mice
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Bennett, M.S.; Akkina, R. Gene Therapy Strategies for HIV/AIDS: Preclinical Modeling in Humanized Mice. Viruses 2013, 5, 3119-3141.

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