Structure-Activity Analysis and Cell-Based Optimization of Human Galactokinase Inhibitors

[摘要]：Classic galactosemia is a rare human disease associated with the accumulation of a toxic level of galactose-1-phosphate (gal-1P) caused by the inherited deficiency of galactose-1-phosphate uridyltransferase (GALT) activity. To reduce the toxic level of gal-1P in patients, we have identified, via high-throughput screening, over 200 small molecule GALK inhibitors. We selected a 4-oxo-3,4-dihydro-2H-1,3-thiazine-5-carbonitrile scaffold for further structure-activity relationships characterization, lead optimization with regards to potency, and efficacy to reduce gal-1P accumulation in patient cells.

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