Intra-Articular AAV9 α-L-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I

Table 3

Comparison of baseline and post-necropsy cartilage HS and DS levels in intra-articular AAV9-treated-MPS I canines.

	Baseline	Necropsy	Percent change
Heparan sulfate
Cartilage HS high-dose +2 w
AAV9-eGFP shoulder	0.7	0.75	7.1%
AAV9-eGFP stifle	0.25	0.49	96.0%
AAV9-IDUA shoulder	0.72	0.41	-43.1%
AAV9-IDUA stifle	0.66	0.31	-53.0%
Cartilage HS high-dose +52 w
AAV9-eGFP shoulder	0.35	9.01	2474%
AAV9-eGFP stifle	0.79	12.7	1508%
AAV9-IDUA shoulder	1.68	5.4	221%
AAV9-IDUA stifle	0.9	8.96	896%
Cartilage HS low-dose +52 w
AAV9-eGFP shoulder	0.42	7.09	1588%
AAV9-eGFP stifle	1.26	7.33	482%
AAV9-IDUA shoulder	0.99	5.99	505%
AAV9-IDUA stifle	1.04	8.82	748%
Dermatan sulfate
Cartilage DS high-dose +2 w
AAV9-eGFP shoulder	2.58	3.3	27.9%
AAV9-eGFP stifle	3.56	4.11	15.4%
AAV9-IDUA shoulder	0.4	2.43	508%
AAV9-IDUA stifle	3.66	4.05	10.7%
Cartilage DS high-dose +52 w
AAV9-eGFP shoulder	0.27	15.69	5711%
AAV9-eGFP stifle	2.36	21.09	794%
AAV9-IDUA shoulder	3.41	14.22	317%
AAV9-IDUA stifle	3.46	14.14	309%
Cartilage DS low-dose +52 w
AAV9-eGFP shoulder	0.29	15.22	5148%
AAV9-eGFP stifle	4.24	13.64	222%
AAV9-IDUA shoulder	0.44	12.33	2702%
AAV9-IDUA stifle	3.14	17.5	457%