Assay Method Information

Assay Name:  IDUA Inhibitory Assay
Description:  The compounds of Example 1 were evaluated on their inhibitory potential against rh-α-IDUA, and their apparent IC50 values were determined by using the fluorogenic substrates 4-methylumbelliferyl-α-iduronide (4-MU-IdoA) and results are shown in Table 1. CIdoA analogues 27a-c with IC50 values in low-micromolar range proved more potent than CIdoADNJ analogues 28a-c, most of which were inactive at 250 μM except 28c. Satisfyingly, a significant reduction of IC50 from 6.7 μM (12) to 1.0 μM (27c) indicated that the conjugation of negatively-charged phosphate group with certain orientation improved the inhibitory potency and then furnished 27c as the most potent IDUA inhibitors in the present disclosure.
Affinity data for this assay
 

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