Table 1.
Outcomes of transactive response DNA-binding protein, 43 kDa (TDP-43)-induced phenotypes in small groups of rats.
| Route | AAV | Promoter | Dose | N | Outcome |
|---|---|---|---|---|---|
| Neonatal i.v. | AAV9 | CBA | 2 × 1012 vg | 1 | Fatal by 2 weeks (Dayton et al., 2013) |
| Neonatal i.v. | AAV9 | Synapsin | 4 × 1012 vg | 3 | Fatal by 2–3 weeks |
| Neonatal i.v. | AAV-PHP.B | CBA | 1.6 × 1012 vg | 2 | Fatal by 10 days, abnormal limb posture |
| Adult i.v. | AAV9 | CBA | 3 × 1013 vg/kg | 3 | Impaired motor function, fatal by 5 weeks post-injection (Jackson et al., 2015b) |
| Adult i.v. | AAV9 | Synapsin | 3 × 1013 vg/kg | 3 | No symptoms up to 6 weeks post-injection |
| Adult i.v. | AAV-PHP.B | Synapsin | 3 × 1013 vg/kg | 3 | Impaired motor function by 2 weeks post-injection, p < 0.05 vs. AAV9 synapsin |
| promoter-TDP-43 on rotarod at 6 weeks | |||||
| Adult i.c.v | AAV9 | CBA | 3.8 × 1012 vg/kg | 1 | Impaired motor function by 2 weeks post-injection |