Summary of rhesus macaques that received regulated Epo AAV vectors
. | AAV transcription factor . | . | AAV target . | . | AAV serotype . | Inducible for duration of study? . | Duration of study . | ||
|---|---|---|---|---|---|---|---|---|---|
| Monkey . | Vector . | GC/kg, × 1012 . | Vector . | GC/kg, × 1012 . | . | . | . | ||
| 95C002 | CMV-TF1 | 4.0 | Z12I-rhEpo-3 | 45.0 | 2 | No | 54 d | ||
| EWP | CMV-TF1 | 7.5 | Z12I-rhEpo-3 | 10.0 | 2 | No | 49 d | ||
| FJX | CMV-TF1 | 7.5 | Z12I-rhEpo-3 | 10.0 | 2 | No | 78 d | ||
| GXF | CMV-TF1 | 7.5 | Z12I-rhEpo-2 | 25.0 | 2 | Yes | 2.0 y | ||
| FFT | CMV-TF1 | 7.5 | Z12I-rhEpo-2 | 25.0 | 2 | Yes* | 2.9 y | ||
| 94B091 | CMV-TF1 | 9.3 | Z12I-rhEpo-2 | 11.5 | 2 | Yes | 6.1 y† | ||
| FTF | CMV-TF1Nc | 20.0 | Z12I-rhEpo-2 | 14.2 | 2 | Yes | 5.5 y† | ||
| AC7B | CMV-TF1Nc | 15.0 | Z12I-rhEpo-2 | 10.0 | 2 | Yes | 5.5 y† | ||
| 97E010 | CMV-TF1Nc | 2.4 | Z12I-rhEpo-2 | 2.4 | 2 | Yes | 1.0 y | ||
| 97E036 | CMV-TF1Nc | 0.7 | Z12I-rhEpo-2 | 0.7 | 2 | Yes | 4.9 y† | ||
| 97E081 | CMV-TF1Nc | 0.2 | Z12I-rhEpo-2 | 0.2 | 2 | Yes | 1.0 y | ||
| 97E102 | CMV-TF1Nc | 1.0 | Z12I-rhEpo-2 | 1.0 | 2 | Yes | 0.5 y | ||
| 97E091 | CMV-TF1Nc | 1.0 | Z12I-rhEpo-2 | 1.0 | 2 | Yes | 4.5 y† | ||
| 97E099 | CMV-TF1INc | 1.0 | Z12I-rhEpo-2 | 1.0 | 2 | Yes | 4.5 y† | ||
| 99E120 | CMV-TF-rhEpo2.3‡ | 1.0 | N/A | 2 | Yes | 1.7 y | |||
| 00E022 | CMV-TF-rhEpo2.3‡ | 0.1 | N/A | 2/1 | Yes | 1.5 y | |||
. | AAV transcription factor . | . | AAV target . | . | AAV serotype . | Inducible for duration of study? . | Duration of study . | ||
|---|---|---|---|---|---|---|---|---|---|
| Monkey . | Vector . | GC/kg, × 1012 . | Vector . | GC/kg, × 1012 . | . | . | . | ||
| 95C002 | CMV-TF1 | 4.0 | Z12I-rhEpo-3 | 45.0 | 2 | No | 54 d | ||
| EWP | CMV-TF1 | 7.5 | Z12I-rhEpo-3 | 10.0 | 2 | No | 49 d | ||
| FJX | CMV-TF1 | 7.5 | Z12I-rhEpo-3 | 10.0 | 2 | No | 78 d | ||
| GXF | CMV-TF1 | 7.5 | Z12I-rhEpo-2 | 25.0 | 2 | Yes | 2.0 y | ||
| FFT | CMV-TF1 | 7.5 | Z12I-rhEpo-2 | 25.0 | 2 | Yes* | 2.9 y | ||
| 94B091 | CMV-TF1 | 9.3 | Z12I-rhEpo-2 | 11.5 | 2 | Yes | 6.1 y† | ||
| FTF | CMV-TF1Nc | 20.0 | Z12I-rhEpo-2 | 14.2 | 2 | Yes | 5.5 y† | ||
| AC7B | CMV-TF1Nc | 15.0 | Z12I-rhEpo-2 | 10.0 | 2 | Yes | 5.5 y† | ||
| 97E010 | CMV-TF1Nc | 2.4 | Z12I-rhEpo-2 | 2.4 | 2 | Yes | 1.0 y | ||
| 97E036 | CMV-TF1Nc | 0.7 | Z12I-rhEpo-2 | 0.7 | 2 | Yes | 4.9 y† | ||
| 97E081 | CMV-TF1Nc | 0.2 | Z12I-rhEpo-2 | 0.2 | 2 | Yes | 1.0 y | ||
| 97E102 | CMV-TF1Nc | 1.0 | Z12I-rhEpo-2 | 1.0 | 2 | Yes | 0.5 y | ||
| 97E091 | CMV-TF1Nc | 1.0 | Z12I-rhEpo-2 | 1.0 | 2 | Yes | 4.5 y† | ||
| 97E099 | CMV-TF1INc | 1.0 | Z12I-rhEpo-2 | 1.0 | 2 | Yes | 4.5 y† | ||
| 99E120 | CMV-TF-rhEpo2.3‡ | 1.0 | N/A | 2 | Yes | 1.7 y | |||
| 00E022 | CMV-TF-rhEpo2.3‡ | 0.1 | N/A | 2/1 | Yes | 1.5 y | |||
The “Duration of study” column indicates the time of the last induction. GC indicates genome copies.
FFT became temporarily refractory to induction during months 3 through 5. Responsivity was restored by month 5 and may have been strengthened by the subsequent administration of sodium phenylbutyrate, a histone deacetylase inhibitor, prior to the next induction at month 10 (“Materials and methods”). Similar treatment of 2 nonresponsive monkeys, EWP and FJX, did not restore inducibility.
Ongoing study.
Single vector.