| Target Validation Information | |||||
|---|---|---|---|---|---|
| Target ID | T65414 | ||||
| Target Name | mRNA of HIV-1 envelope protein | ||||
| Target Type | Successful |
||||
| Action against Disease Model | VRX496 | VRX496 directly interferes with wild-type HIV (wt-HIV) expression via anti-env antisense expression in vector transduced CD4 cells that become infected with wt-HIV. Expression of the anti-HIV antisense env from a HIV vector transcript would target wt-HIV RNA and destroy it, and hence, decrease productive HIV replication from CD4 T cells. | [552277] | Drug Info | |
| References | |||||
| Ref 552277 | 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. | ||||
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