Table 2.
Protocol Comparison
| Wang et al. (2007b)* | This study | |
|---|---|---|
| AAV-micro-dystrophin | ||
| Construct | Canine ΔR4-R23/ΔC | Canine R16-17/H3/ΔC |
| Promoter | CMV | CMV |
| AAV serotype | Type 6 | Type 6, Y445F tyrosine mutant |
| Viral dose (per muscle) | 1×1011 v.g. | 2.85×1012 v.g. |
| Cyclosporin | ||
| Dose | 10 mg/kg/d, oral | 10–20 mg/kg/d, oral |
| Blood level | 100–350 ng/mL | 400–600 ng/mL |
| Duration | −2 day to 18 weeks | −7 day to 4 weeks |
| Mycophenolate | ||
| Dose | 15 mg/kg/d, subcutaneous | 40 mg/kg/d, oral |
| Duration | −2 day to 18 weeks | −7 day to 4 weeks |
| Anti-thymocyte globulin | ||
| Dose | 1 mg/kg/d, subcutaneous | Not used |
| Duration | −2 day to 2 days | |
| Longest time point | ||
| After AAV injection | 30 weeks | 22 weeks |
| After cessation of immune suppression | 12 weeks | 18 weeks |
*
This is different from the protocol used for human micro-dystrophin vector (depicted in Fig 1).