714910-46-0Relevant academic research and scientific papers
Rational design of cell active C2-modified DGJ analogues for the inhibition of human α-galactosidase A (GALA)
Ashmus, Roger A.,Wang, Yang,González-Cuesta, Manuel,King, Dustin T.,Tiet, Ben,Gilormini, Pierre-André,García Fernández, José M.,Ortiz Mellet, Carmen,Britton, Robert,Vocadlo, David J.
supporting information, p. 8057 - 8062 (2021/10/04)
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 inhibitor7c(～100-fold) can act in Fabry patient cells to drive reductions in levels of the disease-relevant glycolipid Gb3.
