Summary of deoxyribozyme gene suppression studies
| Target | Cell | Arms | Modification | Delivery | Activity | Controls | Reference |
|---|---|---|---|---|---|---|---|
| HPV E6 | 3T3 (transient) | 8 /8 | 3′-3′ inversion | DOTAP | 60% suppression E6 RNA | Scrambled | Unpublished |
| c-myc | SMC | 7/7–9/9 | 3′-3′ inversion | DOTAP | 80% suppression cell proliferation | Inactivated | Sun et al., 1999; Cairns et al., 1999 |
| BCR-ABL | BV173 | 8/8–15/15 | 2′O-methyl cap | Lipofectin | Apoptotic morphology | Inactivated & antisense | Warashina et al., 1999 |
| BCR-ABL-luciferase | HeLa (transient) | 8/8–15/15 | 2′O-methyl cap | Lipofectin | 99% suppression luciferase expression | Inactivated & antisense | Warashina et al., 1999 |
| BCR-ABL | K562 | 12 /6 | Phosphorothioate 2-base cap | Cytofectin | 40% protein suppression, 50% cell proliferation | Inactivated | Wu et al., 1999 |
| BCR-ABL | CD34+ CML-bone marrow | 12 /6 | Phosphorothioate 2-base cap | Cytofectin | 53–80% suppression of growth in bcr-abl-positive CFU | Inactivated | Wu et al., 1999 |
| HIV-1 env | HeLa (transient) | 7 /7 | None | Lipofectin | 50% fusion | Irrelevant & antisense | Dash et al., 1998 |
| CCR5 | HeLa (transient) | 7 /7 | None | Lipofectin | 50% fusion | Irrelevant | Goila and Banerjea, 1998 |
| HIV-1 env | U87 | 7 /7 | None | Lipofectamine | 77–81% suppression viral load (p24) | Inactivated | Zhang et al., 1999 |
| huntingtin | HEK-293 (transient) | 8 /8 | 3′-3′ inversion | Lipofectamine | 85% reduction in Huntingtin protein | Inactivated | Yen et al., 1999 |
| NGFI-A | SMC | 9 /9 | 3′-3′ inversion | SuperFect | 75% proliferation | Inactivated & antisense | Santiago et al., 1999 |