Table 2.
Completed HSPC-Based HIV Gene Therapy Trials
| Antiviral Gene (Vector) | ART | GF | E | E-GM (%) | C | I-GM (%) | Outcome |
|---|---|---|---|---|---|---|---|
| tat and tat/rev ribozyme (GV) | yes | NA | NA | NA | NMA | <1 | safe38 |
| MA | |||||||
| tat/vpr ribozyme (GV) | yes | no | 3 | 54 | NMA | <1 | safe, overall lower viral load and higher CD4+ T cells counts75 |
| RRE decoy RNA (GV) | yes | IL-3 | 3 | 17 | NMA | <1 | safe and survival advantage over cells modified with a control vector40 |
| SCF | |||||||
| RevM10 (GV) | yes | SCF | 5 | 24 | NMA | <1 | safe and survival advantage over cells modified with a control vector43 |
| TPO | |||||||
| Flt3L | |||||||
| TdRev (GV) | yes | IL-3 | 4 | 80 | RI | <1 | safe207 |
| GM-CSF | |||||||
| Flt3L | |||||||
| tat/rev shRNA, TAR decoy RNA, CCR5 ribozyme (LV) | yes | SCF | 2 | 18 | MA | <1 | safe and survival advantage over unmodified cells39 |
| TPO | |||||||
| Flt3L |
GV, gammaretroviral vector; LV, lentiviral vector; ART, antiretroviral therapy; GF, growth factors; E, ex vivo culture period in days; E-GM, ex vivo level of gene modification; C, conditioning regimen; MA, myeloablative; NMA, non-myeloablative; RI, reduced intensity; I-GM, in vivo level of gene modification.