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

Table 1

Absolute (% of wild-type) serum IDUA enzyme activity of the three animals treated in this study with respect to timing of treatment.

	Time of necropsy	Time pre-/post-intra-articular AAV9 treatment
		-6 w	+2 w	+12 w	+24 w	+18 w	+52 w
High-dose 5E12 vg/joint	+2 w	0.00 (0%)	2.16 (49.5%)
High-dose 5E12 vg/joint	+52 w	0.00 (0%)	n.m.	0.07 (1.6%)	0.11 (2.5%)	0.14 (3.2%)	0.00 (0%)
Low-dose 5E11 vg/joint	+52 w	0.00 (0%)	n.m.	0.00 (0%)	0.02 (0.46%)	0.05 (1.1%)	0.00 (0%)

All animals have undetectable serum enzyme at baseline (6 weeks prior to treatment); enzyme activities approach 50% of normal two weeks following treatment but decline afterwards, eventually returning to baseline at 52 weeks. Wild-type animal serum IDUA enzyme is units/mL. Data were generated from at least three independent experiments and shown as . n.m.: not measured.