Table 1 Characteristics of the different GRMD dogs of this study.
From: Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Group | Dog | Mode of delivery | Injected product and dose | Injected rAAV batch | Age at inclusion/ injection | Follow-up duration after inclusion/injection |
|---|---|---|---|---|---|---|
Group | LR1 | Locoregional | rAAV2/8-Spc5.12- | A.12005.PSR | 4 Months | 3 Months |
LR | LR2 | transvenous | cMD1 | A.12005.PSR | 4 Months | 3 Months |
LR3 | perfusion | 1 × 1013 vg kg−1 | A.12005.PSR | 3.5 Months | 3 Months | |
LR4 | A.12005.PSR | 3.5 Months | 3 Months | |||
Controls | LR C1 | Locoregional | Buffer | NA | 3.5 Months | 3 Months |
LR | LR C2 | transvenous | NA | 4 Months | 3 Months | |
LR C3 | perfusion | NA | 3.5 Months | 3 Months | ||
Group | IV1 | Intravenous | rAAV2/8-Spc5.12- | A.12005.PSR | 2 Months | 24 Months |
IV-A | IV2 | cMD1 | A.12005.PSR | 2.5 Months | 24 Months | |
IV3 | 1 × 1014 vg kg−1 | A.12005.PSR | 2 Months | 7.5 Months | ||
IV4 | A.12005.PSR | 2.5 Months | 8.5 Months | |||
IV5* | A.12005.PSR | 2 Months | 8 Months | |||
Group | IV6 | Intravenous | rAAV2/8-Spc5.12- | A.12005.PSR | 2 Months | 8.5 Months |
IV-B | IV7 | cMD1 | A.12005.PSR | 2 Months | 8.5 Months | |
IV8 | 2 × 1013 vg kg−1 | 14D0332 | 2.5 Months | 6.5 Months | ||
Controls | IV C1 | NA | NA | NA | 1.5 Months | 3 Months |
IV | IV C2 | (untreated) | NA | 3 Months | 4.5 Months | |
IV C3 | NA | 3 Months | 4.5 Months | |||
IV C4 | NA | 3.5 Months | 9 Months | |||
IV C5 | NA | 3 Months | 4 Months | |||
IV C6 | NA | 3 Months | 23 Months | |||
IV C7 | NA | 2 Months | 3 Months | |||
IV C8 | NA | 2.5 Months | 4.5 Months | |||
IV C9 | NA | 2 Months | 7 Months |
