Research Article
Intra-Articular AAV9 α-L-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I
Table 2
Comparison of baseline and postnecropsy synovial HS and DS levels in intra-articular AAV9-treated-MPS I canines.
| | Baseline | Necropsy | Percent change |
| Heparan sulfate | | | | Synovial HS high-dose +2 w | | | | AAV9-eGFP shoulder | 1.87 | 1.64 | -12.3% | AAV9-eGFP stifle | 3.01 | 3.78 | 25.6% | AAV9-IDUA shoulder | 1.95 | 0.14 | -92.8% | AAV9-IDUA stifle | 4.11 | 0.04 | -99.0% | Synovial HS high-dose +52 w | | | | AAV9-eGFP shoulder | 0.83 | 5.14 | 519% | AAV9-eGFP stifle | 4.5 | 19.4 | 331% | AAV9-IDUA shoulder | 1.73 | 1.63 | -5.8% | AAV9-IDUA stifle | 2.79 | 1.25 | -55.2% | Synovial HS low-dose +52 w | | | | AAV9-eGFP shoulder | 1.27 | 4.35 | 243% | AAV9-eGFP stifle | 1.17 | 9.59 | 720% | AAV9-IDUA shoulder | 0.22 | 2.87 | 1205% | AAV9-IDUA stifle | 2.16 | 3.17 | 46.8% | Dermatan sulfate | | | | Synovial DS high-dose +2 w | | | | AAV9-eGFP shoulder | 6.64 | 4.1 | -38.3% | AAV9-eGFP stifle | 12.32 | 5.78 | -53.1% | AAV9-IDUA shoulder | 8.64 | 1.84 | -78.7% | AAV9-IDUA stifle | 12.5 | 0.8 | -93.6% | Synovial DS high-dose +52 w | | | | AAV9-eGFP shoulder | 3.52 | 9.72 | 176% | AAV9-eGFP stifle | 10.13 | 13.1 | 29.3% | AAV9-IDUA shoulder | 1.87 | 4.23 | 126% | AAV9-IDUA stifle | 7.19 | 2.56 | -64.4% | Synovial DS low-dose +52 w | | | | AAV9-eGFP shoulder | 3.46 | 12.69 | 267% | AAV9-eGFP stifle | 4.98 | 8.68 | 74.3% | AAV9-IDUA shoulder | 0.43 | 4.55 | 958% | AAV9-IDUA stifle | 4.33 | 3.81 | -12.0% |
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