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Stem-Cell-Based Gene Therapy for HIV Infection
Viruses 2014, 6(1), 54-68; doi:10.3390/v6010054
Article

CCR5 as a Natural and Modulated Target for Inhibition of HIV

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Received: 2 September 2013; in revised form: 2 December 2013 / Accepted: 11 December 2013 / Published: 30 December 2013
(This article belongs to the Special Issue Gene Therapy for Retroviral Infections)
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Abstract: Human immunodeficiency virus type 1 (HIV-1) infection of target cells requires CD4 and a co-receptor, predominantly the chemokine receptor CCR5. CCR5-delta32 homozygosity results in a truncated protein providing natural protection against HIV infection—this without detrimental effects to the host—and transplantation of CCR5-delta32 stem cells in a patient with HIV (“Berlin patient”) achieved viral eradication. As a more feasible approach gene-modification strategies are being developed to engineer cellular resistance to HIV using autologous cells. We have developed a dual therapeutic anti-HIV lentiviral vector (LVsh5/C46) that down-regulates CCR5 and inhibits HIV-1 fusion via cell surface expression of the gp41-derived peptide, C46. This construct, effective against multiple strains of both R5- and X4-tropic HIV-1, is being tested in Phase I/II trials by engineering HIV-resistant hematopoietic cells.
Keywords: CCR5; C46; gene therapy; HIV; stem cell transplantation CCR5; C46; gene therapy; HIV; stem cell transplantation
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.

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MDPI and ACS Style

Burke, B.P.; Boyd, M.P.; Impey, H.; Breton, L.R.; Bartlett, J.S.; Symonds, G.P.; Hütter, G. CCR5 as a Natural and Modulated Target for Inhibition of HIV. Viruses 2014, 6, 54-68.

AMA Style

Burke BP, Boyd MP, Impey H, Breton LR, Bartlett JS, Symonds GP, Hütter G. CCR5 as a Natural and Modulated Target for Inhibition of HIV. Viruses. 2014; 6(1):54-68.

Chicago/Turabian Style

Burke, Bryan P.; Boyd, Maureen P.; Impey, Helen; Breton, Louis R.; Bartlett, Jeffrey S.; Symonds, Geoff P.; Hütter, Gero. 2014. "CCR5 as a Natural and Modulated Target for Inhibition of HIV." Viruses 6, no. 1: 54-68.


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