Table 1.

Serum and Tissue Arylsulfatase B Activity in Mucopolysaccharidosis VI Mice Treated with Either Gene Therapy or Enzyme Replacement Therapy

Groups of animals	Serum ARSB	Liver ARSB	Spleen ARSB	Kidney ARSB
NR**	21627±576	132±12**	67±6**	220±18**
AF	—	—	—	—
AAV.hARSB (n=12)**	3614±460	82±30*	0.83±0.30	—
rhARSB (n=6)	—	26±5	4.70±0.52	0.30±0.16

AAV, adeno-associated viral vector; AAV.hARSB, MPS VI affected mice treated with 2×10¹² genome copies (gc)/kg of AAV2/8.TBG.hARSB; AF, MPS VI affected controls; ANOVA, analysis of variance; ARSB, arylsulfatase B; MPS, mucopolysaccharidosis; NR, normal controls; rhARSB, MPS VI affected mice treated with 1 mg/kg of rhARSB.

Measurements in tissues were done at the time of sacrifice. Serum ARSB is reported as the mean activity measured over time for each group. ARSB is expressed as pg/mL for sera and nmol/mg protein/hr for tissues. The number of animals within each treated group is indicated inside the table. The number of animals with the NR and AF group varies depending on the assay. Values are represented as mean±SE. Statistical comparisons were made using the one-way ANOVA and the Tukey post hoc test. The p-value vs. AF is ^*≤0.05 and ^**≤0.01. The exact p-values obtained are indicated in the Materials and Methods section.