Ashmus, Roger A. , Wang, Yang , GONZALEZ CUESTA, MANUEL, King, Dustin T. , Tiet, Ben , Gilormini, Pierre-Andre , Garcia Fernandez, Jose M. , Ortiz Mellet, Carmen , Britton, Robert , Vocadlo, David J.
No
Org. Biomol. Chem.
Article
Científica
1
1
3.89
0.832
07/10/2021
000693679500001
We report the rational design and synthesis of C2-modified DGJ analogues to improve the selective inhibition of human GALA over other glycosidases. We prepare these analogues using a concise route from non-carbohydrate materials and demonstrate the most selective inhibitor 7c (similar to 100-fold) can act in Fabry patient cells to drive reductions in levels of the disease-relevant glycolipid Gb3.