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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.
Received: 17 October 2013; in revised form: 18 November 2013 / Accepted: 19 November 2013 / Published: 28 November 2013
Abstract: 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.
Keywords: 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.
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.
Younan, Patrick; Kowalski, John; Kiem, Hans-Peter. 2013. "Genetic Modification of Hematopoietic Stem Cells as a Therapy for HIV/AIDS." Viruses 5, no. 12: 2946-2962.