TABLE 2. Neutralizing Antibodies in Serum and CSF and Vector Persistence.
Subject | Serum NAb Titer* | CSF NAb titer | CSF | CSF | |||
---|---|---|---|---|---|---|---|
pre- screen |
at injection | 4 weeks post inj. |
at injection |
4 weeks post-inj. |
vg at 2 hours post-injection (per mL) |
circulating vg at 2 hrs* |
|
101 | NT | <1:2 (<1:2) | 1:2048 (1:2048) |
NT | NT | NT | NT |
102 | NT | 1:32 (1:128) |
1:1024 (1:4) | NT | NT | NT | NT |
103 | NT | 1:32 (1:128) |
<1:2 (1:1024) | NT | NT | NT | NT |
104 | NT | 1:2 (1:8) | 1:1024 (>1:2048) |
NT | NT | NT | NT |
303 | <1:2 | <1:2 | 1:256 | <1:2 | <1:2 | NT | NT |
304 | <1:2 | <1:2 | >1:2048 | <1:2 | <1:2 | NT | NT |
201 | 1:32 | 1:32 | 1:1024 | <1:2 | <1:2 | <1×104 | <0.0001% |
202 | 1:128 | 1:128 | 1:1024 | <1:2 | 1:4 | 3.31×105 | <0.0001% |
203 | <1:2 | 1:4 | 1:1024 | <1:2 | <1:2 | 8.02×105 | <0.0001% |
204 | <1:2 | <1:2 | 1:256 | <1:2 | <1:2 | 3.30×108 | 0.0007% |
205 | <1:2 | 1:8 | 1:256 | <1:2 | <1:2 | 6.96×105 | <0.0001% |
206 | <1:2 | <1:2 | 1:64 | <1:2 | <1:2 | <1×104 | <0.0001% |
Abbreviations: CSF = cerebrospinal fluid, NAb = neutralizing antibody, inj. = injection, vg = vector genome, hrs = hours, NT = not tested (sample not available)
Values are for NAb titers against AAV9. NAb titers against AAV2.5 are indicated in parentheses when tested.
Assuming 12 mL of CSF per animal and an even distribution of AAV particles throughout the entire 12 mL, circulating vg in the CSF was calculated as the percentage of the total vg injected, per 12 mL (1.5×1011 vg per mL CSF for low dose, 4.6×1011 vg per mL CSF for high dose).