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Fragment Screening Reveals Starting Points for Rational Design of Galactokinase 1 Inhibitors to Treat Classic Galactosemia

[Image: see text] 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 galac...

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Detalles Bibliográficos
Autores principales: Mackinnon, Sabrina R., Krojer, Tobias, Foster, William R., Diaz-Saez, Laura, Tang, Manshu, Huber, Kilian V. M., von Delft, Frank, Lai, Kent, Brennan, Paul E., Arruda Bezerra, Gustavo, Yue, Wyatt W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Chemical Society 2021
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8056384/
https://www.ncbi.nlm.nih.gov/pubmed/33724769
http://dx.doi.org/10.1021/acschembio.0c00498