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. 2011 Jul 5;19(10):1842–1848. doi: 10.1038/mt.2011.130

Figure 3.

Figure 3

Tissue distribution and stability of recombinant human TPP1 (rhTPP1) following intrathecal administration. Animals were given a single dose of TPP1 proenzyme (80 µl of 10 µg/µl enzyme delivered at 10 µl/minute) on Day 0 and sacrificed at the indicated times. TPP1 specific activities are normalized to wild-type levels. Total endogenous wild type TPP1 levels (pmol/organ, mean ± SD, n = 3) are as follows: brain, 30 ± 1.5; liver, 626 ± 89; heart, 5.5 ± 1.4; and kidney, 144 ± 11. Enzyme half-lives in tissues were estimated fitting TPP1 specific activities to a one phase exponential decay model using GraphPad Prism 5.03. Two points in brain, indicated as gray symbols, were considered outliers and omitted from the regression analysis. Half-life best fit values in days (with 95% confidence intervals) were as follows: brain, 3.0 (2.1–5.0); liver 3.3 (2.8–4.0); heart 2.5 (1.8–3.9); kidney 4.5 (3.5–6.1). An independent experiment where eight Tpp1(−/−) animals were dosed twice a week from 4–8 weeks of age with 20 µl, 10 µg/µl rhTPP1 and then killed 4 and 11 days after the final dose yielded the following half-life estimates: brain, 4.4 (3.1–7.3); liver 4.1 (3.0–6.6); heart 4.4 (2.6–15); kidney 5.0 (3.0–15).