Table 1.
Gene therapy clinical trials targeting the major steps in HIV entry.
Steps in entry targeted | Transgene/Payload | Target cell | % gene marked cells at study conclusion | Ref. |
---|---|---|---|---|
CD4 binding | CD4-zeta chimeric T cell receptor | Autologous CD4+ and CD8+ T cells | 0.1% of PBMCs at 1 year | [41] |
CD4 binding | CD4-zeta chimeric T cell receptor | Syngeneic CD4+ and CD8+ T cells | 0.1%–1% of CD4+ and CD8+ T cells at 1 year | [42] |
CD4 binding | CD4-zeta chimeric T cell receptor | Autologous CD4+ and CD8+ T cells | 0.1%–10% of PBMCs at 24 weeks | [43] |
CCR5 binding | shRNA targeting tat/rev, TAR decoy, CCR5 ribozyme | Autologous CD34+ hematopoietic stem cells | 0.01% of whole blood at 18 months | [44] |
CCR5 binding | CCR5-specific Zinc Finger Nuclease | Autologous CD4+ T cells | Completed | [45] |
CCR5 binding | CCR5-specific Zinc Finger Nuclease (dose escalation) | Autologous CD4+ T cells | Ongoing | [46] |
CCR5 binding | CCR5-specific Zinc Finger Nuclease (dose escalation, with cyclophosphamide) | Autologous CD4+ T cells | Ongoing | [47] |
CCR5 binding | CCR5-specific Zinc Finger Nuclease | Autologous hematopoietic stem cells | Ongoing | [48] |
HIV fusion | HIV fusion inhibitory peptide maC46 | Autologous CD4+ T cells | Less than 0.01% of leukocytes after day 7 | [49] |