Table 2.
Tissue GAA activity 6 months after gene therapy
| Tissue | LV-GAAco MOI 7–9 Gy 106 cells | LV-IGF2.GAAco MOI 7–9 Gy 106 cells | LV-GAAco MOI 2–9 Gy 106 cells | LV-IGF2.GAAco MOI 2–9 Gy 106 cells | LV-GAAco MOI 7–6 Gy 106 cells | LV-IGF2.GAAco MOI 7–6 Gy 106 cells | LV-GAAco MOI 2–6 Gy 106 cells | LV-IGF2.GAAco MOI 2–6 Gy 106 cells | LV-GAAco MOI 7–6 Gy 3 × 105 cells | LV-IGF2.GAAco MOI 7–6 Gy 3 × 105 cells | LV-GAAco MOI 2–6 Gy 3 × 105 cells | LV-IGF2.GAAco MOI 2–6 Gy 3 × 105 cells | KO | WT |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Bone marrow | 1,059.01 ± 437.97 | 116.40 ± 28.40 | 390.08 ± 52.86 | 93.31 ± 11.05 | 374.23 ± 166.00 | 24.39 ± 4.14 | 339.49 ± 83.63 | 37.38 ± 7.58 | 247.49 ± 172.07 | 6.75 ± 2.14 | 124.45 ± 36.80 | 16.20 ± 3.83 | 0.78 ± 0.05 | 4.49 ± 0.37 |
| Leukocytes | 1,085.07 ± 481.45 | 67.11 ± 13.14 | 329.69 ± 11.44 | 55.73 ± 7.68 | 378.84 ± 183.18 | 8.44 ± 1.37 | 307.45 ± 35.56 | 16.74 ± 2.03 | 76.35 ± 16.78 | 2.89 ± 0.64 | 144.71 ± 23.13 | 5.39 ± 0.72 | 0.45 ± 0.03 | 1.84 ± 0.214 |
| Heart | 104.48 ± 30.55 | 9.96 ± 0.49 | 32.20 ± 1.42 | 6.31 ± 0.40 | 39.93 ± 7.66 | 2.74 ± 0.15 | 33.45 ± 2.64 | 3.72 ± 0.29 | 21.02 ± 2.90 | 1.68 ± 0.13 | 18.22 ± 1.80 | 2.16 ± 0.09 | 0.94 ± 0.03 | 15.75 ± 1.201 |
| Tibialis anterior | 41.72 ± 2.33 | 7.25 ± 0.27 | 20.04 ± 1.06 | 5.91 ± 0.47 | 21.57 ± 3.32 | 3.76 ± 0.16 | 19.11 ± 1.13 | 3.63 ± 0.20 | 11.53 ± 1.32 | 2.87 ± 0.21 | 12.82 ± 0.89 | 3.14 ± 0.12 | 1.95 ± 0.07 | 5.20 ± 0.195 |
| Quadriceps femoris | 78.96 ± 10.55 | 5.68 ± 0.20 | 41.12 ± 3.40 | 4.65 ± 0.38 | 30.37 ± 5.76 | 3.34 ± 0.08 | 31.91 ± 2.47 | 3.59 ± 0.28 | 16.80 ± 1.89 | 2.68 ± 0.17 | 18.87 ± 2.40 | 3.38 ± 0.22 | 2.17 ± 0.07 | 6.40 ± 0.251 |
| Gastrocnemius | 53.11 ± 6.00 | 4.97 ± 0.21 | 21.07 ± 1.84 | 4.58 ± 0.26 | 20.09 ± 4.47 | 2.46 ± 0.07 | 15.69 ± 1.68 | 2.62 ± 0.15 | 10.25 ± 0.88 | 2.06 ± 0.06 | 11.90 ± 1.12 | 2.45 ± 0.04 | 1.93 ± 0.04 | 6.36 ± 0.224 |
| Diaphragm | 122.93 ± 18.69 | 9.61 ± 1.03 | 48.91 ± 4.34 | 7.43 ± 0.64 | 42.48 ± 9.98 | 4.03 ± 0.12 | 31.97 ± 3.05 | 4.21 ± 0.26 | 18.53 ± 2.08 | 3.11 ± 0.18 | 23.66 ± 2.15 | 3.78 ± 0.12 | 2.29 ± 0.09 | 9.19 ± 0.322 |
| Cerebrum | 5.11 ± 0.21 | 2.02 ± 0.06 | 3.47 ± 0.42 | 1.85 ± 0.05 | 2.25 ± 0.10 | 1.85 ± 0.02 | 2.49 ± 0.08 | 1.90 ± 0.07 | 2.18 ± 0.06 | 1.80 ± 0.04 | 2.16 ± 0.11 | 1.83 ± 0.03 | 1.61 ± 0.03 | 18.74 ± 0.942 |
| Cerebellum | 7.62 ± 0.52 | 2.55 ± 0.08 | 4.62 ± 0.37 | 2.44 ± 0.14 | 3.37 ± 0.20 | 1.96 ± 0.05 | 3.53 ± 0.20 | 1.99 ± 0.07 | 2.78 ± 0.09 | 2.02 ± 0.06 | 2.99 ± 0.21 | 1.93 ± 0.09 | 2.00 ± 0.04 | 15.46 ± 0.735 |
Activity is expressed in nmol/h/mg of total protein.