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%