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Development of Hematopoietic Stem Cell Based Gene Therapy for HIV-1 Infection: Considerations for Proof of Concept Studies and Translation to Standard Medical Practice

Genetic Modification of Hematopoietic Stem Cells as a Therapy for HIV/AIDS

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA
Department of Medicine, University of Washington, Seattle, WA 98195, USA
Author to whom correspondence should be addressed.
Viruses 2013, 5(12), 2946-2962;
Received: 17 October 2013 / Revised: 18 November 2013 / Accepted: 19 November 2013 / Published: 28 November 2013
(This article belongs to the Special Issue Gene Therapy for Retroviral Infections)
The combination of genetic modification and hematopoietic stem cell (HSC) transplantation may provide the necessary means to develop an alternative treatment option to conventional antiretroviral therapy. As HSCs give rise to all hematopoietic cell types susceptible to HIV infection, modification of HSCs is an ideal strategy for the development of infection-resistant immune cell populations. Although promising results have been obtained in multiple animal models, additional evidence is needed to convincingly demonstrate the feasibility of this approach as a treatment of HIV-1 infected patients. Here, we review the potential of HSC transplantation and the recently identified limitations of this approach. Using the Berlin Patient as a model for a functional cure, we contrast the confines of autologous versus allogeneic transplantation. Finally, we suggest that although autologous, gene-modified HSC-transplantation may significantly reduce plasma viremia, reaching the lower detection limits currently obtainable through daily HAART will remain a challenging endeavor that will require innovative combinatorial therapies. View Full-Text
Keywords: HIV/AIDS; Gene Therapy; Stem Cell Transplantation HIV/AIDS; Gene Therapy; Stem Cell Transplantation
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MDPI and ACS Style

Younan, P.; Kowalski, J.; Kiem, H.-P. Genetic Modification of Hematopoietic Stem Cells as a Therapy for HIV/AIDS. Viruses 2013, 5, 2946-2962.

AMA Style

Younan P, Kowalski J, Kiem H-P. Genetic Modification of Hematopoietic Stem Cells as a Therapy for HIV/AIDS. Viruses. 2013; 5(12):2946-2962.

Chicago/Turabian Style

Younan, Patrick, John Kowalski, and Hans-Peter Kiem. 2013. "Genetic Modification of Hematopoietic Stem Cells as a Therapy for HIV/AIDS" Viruses 5, no. 12: 2946-2962.

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