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. Author manuscript; available in PMC: 2015 Apr 28.
Published in final edited form as: Sci Transl Med. 2014 Apr 9;6(231):231ra48. doi: 10.1126/scitranslmed.3007733

Table 2.

βgal activity in brain, spinal cord, CSF, and liver of AAV-treated and untreated GM1 cats.

Fold-normal βgal specific activity, mean (s.d)
Region Block Short term, full doseA AAVrh8* Short term, full doseB AAV1* Long term, 1/10th doseC AAVrh8 Long term, full doseD AAVrh8 Long term, full doseE AAV1 GM1 no tx
Cerebrum a 2.7 (0.85) 4.1 (4.4) 0.48 (0.24) 0.96 (0.37) 0.48 0.00 (0.00)
b 1.8 (0.62) 2.3 (2.4) 0.42 (0.11) 0.71 (0.30) 0.42 0.00 (0.00)
c 1.7 (0.56) 2.2 (2.0) 0.63 (0.57) 0.56 (0.17) 0.61 0.01 (0.01)
d 1.7 (0.70) 2.6 (1.1) 0.62 (0.40) 0.78 (0.27) 1.2 0.02 (0.07)
e 1.1 (0.56) 1.4 (1.1) 0.33 (0.16) 0.78 (0.35) 0.82 0.05 (0.02)
Cerebellum f 1.5 (1.2) 3.0 (1.2) 0.55 (0.90) 2.9 (1.5) 0.40 0.10 (0.04)
g 2.2 (1.8) 4.2 (2.1) 0.33 (0.23) 2.5 (0.96) 0.33 0.04 (0.02)
h 4.1 (2.6) 1.7 (1.1) 0.27 (0.34) 1.4 (1.6) 0.30 0.04 (0.03)
Spinal cord i 2.0 (1.5) 1.0 (0.38) 0.00 (0.00) 0.74 (0.65) 0.07 0.00 (0.00)
j 1.6 (1.4) 0.85 (0.60) 0.03 (0.06) 1.2 (0.93) 0.04 0.00 (0.00)
k 1.9 (1.0) 0.89 (0.28) 0.05 (0.08) 1.5 (1.1) 0.10 0.03 (0.05)
l 3.2 (2.5) 1.0 (0.46) 0.07 (0.09) 0.93 (0.62) 0.00 0.03 (0.06)
m 2.1 (1.4) 2.2 (1.9) 0.07 (0.13) 0.75 (0.68) 0.05 0.00 (0.00)
n 2.3 (1.1) 1.8 (1.5) 0.09 (0.15) 1.3 (0.98) 0.05 0.04 (0.07)
o 4.8 (3.1) 4.2 (3.2) 0.38 (0.61) 6.6 (8.5) 0.17 0.01 (0.02)
CSF N/A 28 (20) 42 (28) 2.7 (1.3) 32 (42) 8.3 0.03 (0.08)
Liver N/A 0.38 (0.25) 0.24 (0.16) 0.17 (0.11) 0.20 (0.19) 0.08 0.05 (0.01)
*

βgal activity was not significantly different between AAVrh8 (n = 4) and AAV1 (n = 3) cohorts at the 16 week time point (a–h and i–o, P ≥ 0.22 for each block; CSF, P = 0.19; liver, P = 0.43).

A

n = 4; βgal specific activity was significantly higher than untreated GM1 cats (n = 4) in a-h and i-o (P ≤ 0.015 for each block), CSF (P = 0.026, n = 3, no sample available for 8-1526), and liver (P = 0.015).

B

n = 3; βgal specific activity was significantly higher than untreated GM1 cats (n = 4) in a-h and i-o (P ≤ 0.026 for each block), CSF (P = 0.026), and liver (P = 0.026).

C

n = 4; βgal specific activity was significantly higher than untreated GM1 cats (n = 4) in a-e, g, and h (P ≤ 0.021 for each block), CSF (P = 0.015), and liver (P = 0.015), but not in f (P = 0.15) or i-o (P ≥ 0.20 for each block).

D

n = 3; βgal specific activity was significantly higher than untreated GM1 cats (n = 4) in a-h, j-l, and n-o (P ≤ 0.025 for each block), as well in CSF (P = 0.025), but not in blocks i and m (P = 0.061) or liver (P = 0.054).

E

Not enough subjects currently available for statistical analysis.

The Wilcoxon signed-rank test was used for statistical comparisons.

Abbreviations: no tx = no treatment; N/A = not applicable.