Table 1.
AAV minidystrophin gene transfer in young and adult mdx mice
| Animals* and vectors | n | Age at vector injection | Months post injection | % Dystrophin-positive fibers | % Central nuclei† |
|---|---|---|---|---|---|
| mdx + Δ3849 | 4 | 10 days | 3 | 56 ∼ 88 | 1.02 (72/7,098) |
| mdx + Δ3990 | 4 | 10 days | 3 | 50 ∼ 80 | 0.99 (56/5,652) |
| mdx + Δ4173 | 4 | 10 days | 3 | 15 ∼ 25 | 0.93 (26/2,791) |
| mdx + Δ3849 | 4 | 10 days | 6 | 40 ∼ 60 | 2.80 (51/1,824) |
| mdx + Δ3990 | 2 | 10 days | 6 | 35 ∼ 45 | 2.30 (34/1,478) |
| mdx + Δ3849 | 2 | 50 days | 2 | 35 ∼ 50 | 34.76 (510/1,467) |
| mdx + Δ3990 | 2 | 50 days | 2 | 35 ∼ 40 | 34.18 (685/2,004) |
| mdx + Δ3849 | 4 | 50 days | 4 | 20 ∼ 25 | 44.24 (615/1,390) |
| mdx + Δ3990 | 4 | 50 days | 4 | 20 ∼ 30 | 46.18 (695/1,505) |
| C57/B10 | 4 | No injection | N/A | 100 | 1.45 (56/3,860) |
| mdx | 4 | No injection | N/A | <1 | 75.4 (238/3,160) |
N/A, Not applicable.
*
Untreated control mdx and C57/B10 mice were about 3 months old at the endpoints of experiments. AAV vectors were driven by MCK promoter.
†
All numbers were collected from dystrophin-positive myofibers that were photographed after IF staining and DAPI counterstaining, except in untreated mdx mice, which had extensive central nucleation and very few dystrophin-positive revertant myofibers.