Identification of pharmacological chaperones as potential therapeutic agents to treat phenylketonuria
J. Clin. Invest. Angel L. Pey, et al. 118:2858 doi:10.1172/JCI34355 [
Go to this article.]
Figure 3Effect of the hit compounds on the activity of recombinant WT-PAH. Activity was measured with 100 μM
l-Phe and 10 μM BH
4 (
A) or 1 mM
l-Phe and 200 μM BH
4 (
B), in the presence of 100 μM of each compound in 2.5% DMSO (also present in control sample [C]). Samples were preincubated (black bars) or not preincubated (white bars) with the corresponding
l-Phe concentration for 5 minutes at 25°C prior to the addition of compounds and of BH
4 to start activity measurements. Data are mean ± SD of triplicate experiments.
