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  • VRX496 is the first and only lentiviral vector therapy approved by the FDA for clinical trials, according to VIRxSYS. The backbone of VRX496 consists of a genetically engineered version of HIV in which all the infectious components are removed and replaced with the therapeutic payload—a long antisense sequence that targets the HIV envelope protein and cripples the virus. (en)
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  • # Humeau LM, Binder GK, Lu X, Slepushkin V, Merling R, Echeagaray P, Pereira M, Slepushkina T, Barnett S, Dropulic LK, Carroll R, Levine BL, June CH, Dropulic B: Efficient lentiviral vector-mediated control of HIV-1 replication in CD4 lymphocytes from diverse HIV+ infected patients grouped according to CD4 count and viral load. Mol Ther. 2004 Jun;9(6):902-13. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/15194057 # MacGregor RR: Clinical protocol. A phase 1 open-label clinical trial of the safety and tolerability of single escalating doses of autologous CD4 T cells transduced with VRX496 in HIV-positive subjects. Hum Gene Ther. 2001 Nov 1;12(16):2028-9. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/11727736 (en)
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  • investigational (en)
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  • HIV infection (en)
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  • VRX496 (en)
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  • VRX496 is an autologous therapy that uses a patient’s own cells. Clinical sites collect white blood cells from individual patients by apheresis. VIRxSYS scientists purify the white blood cells to isolate CD4 T cells, which are transduced with VRX496. The genetically modified cells are expanded and reinfused into the patient. When HIV enters CD4 T cells to replicate, the antisense payload of VRX496 is transcribed, which binds the virus and destroys it. The goal is to repopulate a patient’s immune system with genetically engineered cells that promote immunity against HIV and prevent the progression to AIDS. Although not a cure, VRX496 could improve the quality of life for HIV patients by bringing viral loads down to low levels. This approach could slow or even reverse a patient’s progression to full-blown AIDS. (en)
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