Target Validation Information
TTD ID T65414
Target Name Human immunodeficiency virus Envelope messenger RNA (HIV env mRNA)
Type of Target
Successful
Action against Disease Model VRX496 Drug Info 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. [1]
References
REF 1 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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