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Stem-Cell-Based Gene Therapy for HIV Infection
Article

CCR5 as a Natural and Modulated Target for Inhibition of HIV

1
Calimmune, Inc., Los Angeles, California, CA 90024, USA
2
Institute of Transfusion Medicine and Immunology, Medical Faculty Mannheim, Heidelberg University, German Red Cross Blood Service Baden-Württemberg-Hessen, 68167 Mannheim, Germany
3
Cellex GmbH, 01307 Dresden, Germany
*
Author to whom correspondence should be addressed.
Viruses 2014, 6(1), 54-68; https://doi.org/10.3390/v6010054
Received: 2 September 2013 / Revised: 2 December 2013 / Accepted: 11 December 2013 / Published: 30 December 2013
(This article belongs to the Special Issue Gene Therapy for Retroviral Infections)
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. View Full-Text
Keywords: CCR5; C46; gene therapy; HIV; stem cell transplantation CCR5; C46; gene therapy; HIV; stem cell transplantation
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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. https://doi.org/10.3390/v6010054

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. https://doi.org/10.3390/v6010054

Chicago/Turabian Style

Burke, Bryan P., Maureen P. Boyd, Helen Impey, Louis R. Breton, Jeffrey S. Bartlett, Geoff P. Symonds, and Gero Hütter. 2014. "CCR5 as a Natural and Modulated Target for Inhibition of HIV" Viruses 6, no. 1: 54-68. https://doi.org/10.3390/v6010054

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