Table 3.
Summary of factors delivery with gene therapy
| Agent | Model | Administration | Dose | Start (days) | Onset change (%) | Survival change (%) | Reference |
|---|---|---|---|---|---|---|---|
| GDNF plasmid | SOD1G93A | Intramuscular | 50 mg plasmid fortnightly | 63 | ↑ | ns | |
| AV-CT-1 | SOD1G93A | Intramuscular | 1 × 108 units | 5–6 | ↑21 | ↑8 | |
| AV-GDNF | SOD1G93A | Intramuscular | 5 × 109 units | 5–7 | ↑7 | ↑12 | |
| AAV-Bcl-`2 | SOD1G93A | Intramuscular | ~1 × 108 units | 35 | ↑8 | Not significant | |
| AAV-GDNF | SOD1G93A | Intramuscular | 1 × 1011 units | 63 | ↑13 | ↑14 | |
| AAV-GDNF | SOD1G93A | Intramuscular | 1 × 1010 units | 60 | ↑18 | ↑9 | |
| AAV-GDNF | SOD1G93A | Intramuscular | 1 × 1010 units | 90 | nd | ↑6 | |
| AAV-Follistatin | SOD1G93A | Intramuscular | 1 × 1011 units | 40 | nd | Not significant | Miller et al. [27] |
| AAV-IGF-1 | SOD1G93A | Intramuscular | 1 × 1010 units | 60 | ↑34 | ↑30 | |
| AAV-IGF-1 | SOD1G93A | Intramuscular | 1 × 1010 units | 90 | nd | ↑18 | |
| AAV-IGF-1 | SOD1G93A | Intraspinal | 5 × 1010 units | 60 | ↑20 | ↑10 | Lepore et al. [80] |
| EIAV-VEGF | SOD1G93A | Intramuscular | 1 × 108 units | 21 | ↑30 | ↑30 | |
| EIAV-VEGF | SOD1G93A | Intramuscular | 1 × 108 units | 90 | nd | ↑18 | |
| PNA GluR3 | SOD1G93A | Intraperitoneal | 2.5 mg/kg, 3 times a week | 50 | ↑9 | ↑9 | Rembach et al. [42] |
| PNA p75NTR | SOD1G93A | Intraperitoneal | 2.5 mg/kg, 3 times a week | 60 | ↑10 | ↑8 | Turner et al. [41] |
| siRNA Fas | SOD1G93A | Intraspinal | 1 mg/kg days | 90 | ↑ | ↑14 | Locatelli et al. [43] |