Fragment screening reveals starting points for rational design of galactokinase 1 inhibitors to treat classic galactosemia

Classic galactosemia is caused by loss-of-function mutations in galactose-1-phosphate uridylyltransferase (GALT) that lead to toxic accumulation of its substrate, galactose-1-phosphate. One proposed therapy is to inhibit the biosynthesis of galactose-1-phosphate, catalyzed by galactokinase 1 (GALK1)...

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Bibliographic Details
Main Authors: Mackinnon, SR, Krojer, T, Foster, WR, Diaz-Saez, L, Tang, M, Huber, KVM, von Delft, F, Lai, K, Brennan, PE, Arruda Bezerra, G, Yue, WW
Format: Journal article
Language:English
Published: American Chemical Society 2021